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An insurer has turned down Sara Aldrich's bid for coverage for Trikafta, described by an advocate as the single greatest innovation in cystic fibrosis history.

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Sara Aldrich believes you cant put a price on life.

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But others are doing just that, and the value theyve assigned to hers is less than the cost of a recently approved drug, Trikafta, that could greatly improve and lengthen it.

Aldrich, a 23-year-old student and Stittsville resident, has cystic fibrosis, a progressive, degenerative multi-system disease that affects mainly the lungs and digestive system by creating a buildup of thick mucus, causing respiratory ailments and making digestion and the absorption of nutrients difficult.

There is no known cure, and in 2018 half of the deaths in Canada from CF involved people younger than 33.

Diagnosed at 16 months, Aldrich led an active lifestyle throughout her childhood and teens, playing competitive soccer. She fully recovered from a lung collapse when she was in Grade 11, but well recalls the warning a doctor at the hospital once offered, telling her to prepare for her death.

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Meanwhile, despite a rigorous daily regimen aimed at slowing further decline, Aldrichs lung function has dropped from between 80 and 90 per cent three years ago to the low 40s today. At this rate, she estimates shell be on the lung-transplant list within two years.

My dream is to run a marathon, she says, but, as of right now, even walking up the stairs, I usually cough so much that I throw up. I dont sleep through the night anymore. Im always exhausted. Im seeing myself decline very, very quickly, and we dont know how to put a stop to it. Ive had infection after infection.

Last June, however, CF patients in Canada were encouraged when Health Canada approved Trikafta, a drug that Kelly Grover, president and CEO of Cystic Fibrosis Canada, described as the single greatest innovation in cystic fibrosis history (with) the power to transform the lives of thousands of Canadians.

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While not a cure, Cystic Fibrosis Canadas Kim Steele says, its by far the closest thing yet.

We are seeing people who were on the lung transplant list go off the list and return to work or be able to raise children. In the U.S., theres been a baby boom because people with CF are in a spot where theyre thinking, Im healthy enough to have a child and Im going to be around for a while to care for that child.

Trikafta, she adds, has been transformational.

With an annual list price of about $300,000, though, the drug would otherwise be out of reach of all but the wealthiest, so many welcomed the news in September that Ontario was adding Trikafta to the provincial drug program.

Our government has taken urgent action to ensure all cystic fibrosis patients will have more timely access to the effective and life-changing treatments they need, Minister of Health Christine Elliott said.

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Because shes still in school, Aldrich was able to apply for coverage through her mothers Ontario Teachers Insurance Plan, or OTIP, benefits for which are paid through Manulife. But OTIP, and many other groups that administer benefits for employee health plans, employs FACET, an independent team of clinical pharmacists that reviews and approves, or denies, requests for specialty drug therapies.

Two weeks ago, Aldrich received an email from FACET, saying her application was denied. The letter cited a recent recommendation by the Canadian Drug Expert Committee (CDEC) of the Canadian Agency for Drugs and Technologies in Health (CADTH), indicating Trikafta was not cost-effective.

A 90% price reduction would be required for Trikafta to reach acceptable costeffectiveness thresholds, the letter read. As a result, the group benefits plan is unable to provide coverage for Trikafta at this time.

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Aldrich is, understandably, devastated.

You cant put a price on someones life, and right now it is a matter of life and death.

Imagine being told that theres this amazing drug thats going to save you and then have it ripped away from you.

Steele, meanwhile, is upset, noting that the recommendation from CADTH is a non-binding one intended for the countrys public payers, such as federal and provincial health plans, which take into consideration such factors as long-term hospitalizations and transplants.

Our governments pay for those, and they have decided this drug is worth funding because it has greater value than relegating people to hospital beds for the rest of their lives and shortening their lives, she says.

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So, when a private insurer has not moved to cover this, its a real problem because what theyre saying, essentially, is that the government can fund it and they dont have to, and thats shameful.

Additionally, Steele notes, governments typically dont pay the full list price for a drug, instead joining forces to negotiate a better, private deal.

Our governments were able to negotiate that price down, probably not 90 per cent, but somewhere in the middle. And, if our public payers can do that, the private insurers that charge premiums can. Its a total smokescreen, and people are paying into these private plans and not getting the benefits, not getting the drugs they need.

Steele admits theres no easy fix to what is a multi-layered problem, but points to the model used in Quebec, where mandated private insurance led to a cooperative risk-share model.

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She also suggests that private payers should be at the table with public ones when negotiating drug prices. Everybody has a bit to pay here, and, if we all put our money in the same pot, we can get better results and people can get the medicine that they need faster and in a more fulsome way.

Mike Sullivan, CEO of Cubic Health, which administers FACET, says the practice of provinces jointly negotiating private agreements with pharmaceutical companies, without insurance companies at the table, creates a two-tier pricing system for expensive drugs such as Trikafta.

(It) creates a very unbalanced playing field, he said in an email. The benefit plans have limited funding to provide all benefits (not just drug), and, if they have to pay multiples of what the public plans have to pay, they cannot cover the same breadth of medications.

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In the meantime, Aldrich weighs her options while her lung function worsens. She could apply to the provinces OHIP+ plan, but would have to forego any other coverage under her mothers benefits plan, while any coverage she might be approved for would end next year, when she turns 25. That would at least fill a gap, during which she hopes insurance companies will realize how important Trikafta is to CF patients.

Theres also the possibility that she and her brother Chris, who also has CF, might be able to get Trikafta through the Ontario Disability Support Program.

Weve been working pretty hard for me to get Trikafta because its pretty much my last hope, she says.

Without Trikafta, I dont really plan for my future because theres this fear that I wont have a future. But, with Trikafta, I see myself accomplishing the goals that Ive always set out for myself. Ive always had a passion to teach, and Ive always wanted a family that is my absolute dream. I know that, with Trikafta, I would be able to start a family and have a house and everything that you dream of as someone without CF. I see the potential in my life and living a long life.

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23-year-old Stittsville student learns life is worth less than the drug that could save it - Ottawa Citizen

Dublin, Nov. 10, 2021 (GLOBE NEWSWIRE) -- The "Ammonium Nitrate Market Research Report by Application, End-user, and Region - Global Forecast to 2026 - Cumulative Impact of COVID-19" report has been added to ResearchAndMarkets.com's offering.

The Global Ammonium Nitrate Market size was estimated at USD 17.70 billion in 2020, is expected to reach USD 18.58 billion in 2021, and projected to grow at a CAGR of 5.33% reaching USD 24.17 billion by 2026.

Market Statistics

The report provides market sizing and forecast across five major currencies - USD, EUR GBP, JPY, and AUD. It helps organization leaders make better decisions when currency exchange data is readily available. In this report, the years 2018 and 2019 are considered historical years, 2020 as the base year, 2021 as the estimated year, and years from 2022 to 2026 are considered the forecast period.

Market Segmentation & Coverage

This research report categorizes the Ammonium Nitrate to forecast the revenues and analyze the trends in each of the following sub-markets:

Based on Application, the market was studied across Explosives and Fertilizers.

Based on End-user, the market was studied across Agriculture, Civil and Commercial Explosives, Military Explosives, and Mining and Quarrying.

Based on Region, the market was studied across Americas, Asia-Pacific, and Europe, Middle East & Africa. The Americas is further studied across Argentina, Brazil, Canada, Mexico, and United States. The United States is further studied across California, Florida, Illinois, New York, Ohio, Pennsylvania, and Texas. The Asia-Pacific is further studied across Australia, China, India, Indonesia, Japan, Malaysia, Philippines, Singapore, South Korea, Taiwan, and Thailand. The Europe, Middle East & Africa is further studied across France, Germany, Italy, Netherlands, Qatar, Russia, Saudi Arabia, South Africa, Spain, United Arab Emirates, and United Kingdom.

Competitive Strategic Window

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The Competitive Strategic Window analyses the competitive landscape in terms of markets, applications, and geographies to help the vendor define an alignment or fit between their capabilities and opportunities for future growth prospects. It describes the optimal or favorable fit for the vendors to adopt successive merger and acquisition strategies, geography expansion, research & development, and new product introduction strategies to execute further business expansion and growth during a forecast period.

FPNV Positioning Matrix

The FPNV Positioning Matrix evaluates and categorizes the vendors in the Ammonium Nitrate Market based on Business Strategy (Business Growth, Industry Coverage, Financial Viability, and Channel Support) and Product Satisfaction (Value for Money, Ease of Use, Product Features, and Customer Support) that aids businesses in better decision making and understanding the competitive landscape.

Market Share Analysis

The Market Share Analysis offers the analysis of vendors considering their contribution to the overall market. It provides the idea of its revenue generation into the overall market compared to other vendors in the space. It provides insights into how vendors are performing in terms of revenue generation and customer base compared to others. Knowing market share offers an idea of the size and competitiveness of the vendors for the base year. It reveals the market characteristics in terms of accumulation, fragmentation, dominance, and amalgamation traits.

Competitive Scenario

The Competitive Scenario provides an outlook analysis of the various business growth strategies adopted by the vendors. The news covered in this section deliver valuable thoughts at the different stage while keeping up-to-date with the business and engage stakeholders in the economic debate. The competitive scenario represents press releases or news of the companies categorized into Merger & Acquisition, Agreement, Collaboration, & Partnership, New Product Launch & Enhancement, Investment & Funding, and Award, Recognition, & Expansion. All the news collected help vendor to understand the gaps in the marketplace and competitor's strength and weakness thereby, providing insights to enhance product and service.

Company Usability Profiles

The report profoundly explores the recent significant developments by the leading vendors and innovation profiles in the Global Ammonium Nitrate Market, including Austin Powder Holdings Company, CF Industries Holdings, Inc., Enaex S.A, EuroChem Group AG, Incitec Pivot Limited, Neochim PLC, Orica Limited., OSTCHEM Holding AG, San Corporation, and URALCHEM Holding P.L.C.

The report provides insights on the following pointers:1. Market Penetration: Provides comprehensive information on the market offered by the key players2. Market Development: Provides in-depth information about lucrative emerging markets and analyze penetration across mature segments of the markets3. Market Diversification: Provides detailed information about new product launches, untapped geographies, recent developments, and investments4. Competitive Assessment & Intelligence: Provides an exhaustive assessment of market shares, strategies, products, certification, regulatory approvals, patent landscape, and manufacturing capabilities of the leading players5. Product Development & Innovation: Provides intelligent insights on future technologies, R&D activities, and breakthrough product developments

The report answers questions such as:1. What is the market size and forecast of the Global Ammonium Nitrate Market?2. What are the inhibiting factors and impact of COVID-19 shaping the Global Ammonium Nitrate Market during the forecast period?3. Which are the products/segments/applications/areas to invest in over the forecast period in the Global Ammonium Nitrate Market?4. What is the competitive strategic window for opportunities in the Global Ammonium Nitrate Market?5. What are the technology trends and regulatory frameworks in the Global Ammonium Nitrate Market?6. What is the market share of the leading vendors in the Global Ammonium Nitrate Market?7. What modes and strategic moves are considered suitable for entering the Global Ammonium Nitrate Market?

Key Topics Covered:

1. Preface

2. Research Methodology

3. Executive Summary

4. Market Overview4.1. Introduction4.2. Cumulative Impact of COVID-19

5. Market Dynamics5.1. Introduction5.2. Drivers5.2.1. Need for nitrogen rich fertilizers to increase the agriculture produce from limited arable land5.2.2. Proliferation in construction and mining sector5.2.3. Robust infrastructure development in developing economies5.3. Restraints5.3.1. Volatile price of nitric acid5.4. Opportunities5.4.1. Emerging expansion and remodeling of older production facilities along with manufacture of new ammonium nitrate plants5.4.2. Demand for explosives in defense sector5.5. Challenges5.5.1. Numerous environmental regulations for storage, transport, and packaging

6. Ammonium Nitrate Market, by Application6.1. Introduction6.2. Explosives6.3. Fertilizers

7. Ammonium Nitrate Market, by End-user7.1. Introduction7.2. Agriculture7.3. Civil and Commercial Explosives7.4. Military Explosives7.5. Mining and Quarrying

8. Americas Ammonium Nitrate Market8.1. Introduction8.2. Argentina8.3. Brazil8.4. Canada8.5. Mexico8.6. United States

9. Asia-Pacific Ammonium Nitrate Market9.1. Introduction9.2. Australia9.3. China9.4. India9.5. Indonesia9.6. Japan9.7. Malaysia9.8. Philippines9.9. Singapore9.10. South Korea9.11. Taiwan9.12. Thailand

10. Europe, Middle East & Africa Ammonium Nitrate Market10.1. Introduction10.2. France10.3. Germany10.4. Italy10.5. Netherlands10.6. Qatar10.7. Russia10.8. Saudi Arabia10.9. South Africa10.10. Spain10.11. United Arab Emirates10.12. United Kingdom

11. Competitive Landscape11.1. FPNV Positioning Matrix11.1.1. Quadrants11.1.2. Business Strategy11.1.3. Product Satisfaction11.2. Market Ranking Analysis11.3. Market Share Analysis, by Key Player11.4. Competitive Scenario11.4.1. Merger & Acquisition11.4.2. Agreement, Collaboration, & Partnership11.4.3. New Product Launch & Enhancement11.4.4. Investment & Funding11.4.5. Award, Recognition, & Expansion

12. Company Usability Profiles12.1. Austin Powder Holdings Company12.2. CF Industries Holdings, Inc.12.3. Enaex S.A12.4. EuroChem Group AG12.5. Incitec Pivot Limited12.6. Neochim PLC12.7. Orica Limited.12.8. OSTCHEM Holding AG12.9. San Corporation12.10. URALCHEM Holding P.L.C.

13. Appendix

For more information about this report visit https://www.researchandmarkets.com/r/jdfmwk

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Insights on the Ammonium Nitrate Global Market to 2026 - Featuring CF Industries, Enaex and Neochim Among Others - Yahoo Finance

Calgarians are invited to enjoy a safe and fun-filled holiday mini-putt experience presented by Cadillac Fairview

CALGARY, AB, Nov. 4, 2021 /CNW/ - Cadillac Fairview (CF) is continuing with its traditions of spirited and engaging community experiences to deliver holiday cheer to Calgarians by transforming a portion of its parking lot into Frozen Fairways, an outdoor activity that merges two sports that are popular among Canadians: golf and hockey. From November 17 to 21, Calgarians are encouraged to safely take part in the five day holiday mini-putt experience that replaces a putter, greens and balls with a hockey stick and puck and artificial ice. Frozen Fairways features nine-holes, each with exciting Canadian twists including beloved activities like hockey and curling, and cultural staples like icing fishing and sugar shacks. Participants can reserve their time slot by pre-booking online. Tickets are $5 each and will go on sale starting on November 5. All proceeds from ticket sales will benefit local charities.

CF Chinook Centre logo (CNW Group/Cadillac Fairview Corporation Limited)

Additionally, Cadillac Fairview and RBC Loyalty & Rewards have come together to offer RBC credit cards exclusive perks, including advance to tickets, waived entry fees and more.

CF Chinook Centre continues to follow Alberta health guidelines with safety as a top priority for its community of guests and employees by integrating extensive health and safety measures in all of its holiday programming. As an outdoor event, the Frozen Fairways experience has enforced physical distancing measures, pre-screening (at home and onsite), regular and frequent sanitation procedures and minimized contact wherever possible. Additionally, to limit the number of guests visiting the activation and to minimize crowding, the experience is only accessible to guests via online registration. Each reservation includes one round of mini putt at the Frozen Fairways and groups will face off ten minutes apart. All guests are encouraged to wear masks and practice physical distancing on and off the course.

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Calgarians of all ages are welcome to participate in this shareworthy, festive and limited-time activity bringing family and friends together in a reimagined, safe and enjoyable way.

What:

CF Frozen Fairways

Where:

CF Chinook Centre Wednesday, November 17 to Sunday, November 21, 2021

Interview Opp:

Interview with Paige O'Neill, General Manager, CF Chinook Centre

Onsite Photo Opp:

Calgarians celebrate the holiday season at CF Chinook Centre's magical Frozen Fairways; imagery of Canadian-inspired Frozen Fairways mini-putt course.

For more information, event hours and to make reservations,visit https://shops.cadillacfairview.com/holiday.

About Cadillac FairviewCadillac Fairview (CF) is a globally focused owner, operator, investor, and developer of best-in-class real estate across retail, office, residential, industrial, and mixed-use asset classes. Wholly owned by the Ontario Teachers' Pension Plan, CF manages in excess of $35 billion of assets across the Americas and the United Kingdom, with further expansion planned into Europe and Asia.

Internationally, CF invests in communities with like-minded partners, including Stanhope in the UK, Lincoln Property Company in the U.S., and Multiplan in Brazil. The company's Canadian portfolio comprises 68 landmark properties, including the Toronto-Dominion Centre, CF Toronto Eaton Centre, Tour Deloitte, CF Carrefour Laval, CF Chinook Centre, and CF Pacific Centre.

Continually striving to make a positive impact in communities where it operates by promoting social connection, growth, and a sustainable future, CF's Purpose is Transforming Communities for a Vibrant Tomorrow. Learn more at cadillacfairview.com and follow CF on LinkedIn.

SOURCE Cadillac Fairview Corporation Limited

Cision

View original content to download multimedia: http://www.newswire.ca/en/releases/archive/November2021/04/c8531.html

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CF Chinook Centre Hosts Frozen Fairways Inviting Shoppers to take in New Outdoor Sports Experience from November 17 to 21 - Yahoo Finance

One of Canadas CF-18 Hornet fighter jets will fly over Beardys and Okemasis Cree Nation in honour of Indigenous veterans.

The community plans to hold their National Indigenous Veterans Day ceremony on Nov. 8, with the RCMP fighter jet scheduled to fly by at around 11 a.m.

Typically, RCAF fighters only fly past communities on request. RCAF public affairs officer David Lavallee said they carefully consider each application before committing to a fly over.

We support where we can and where appropriate, Lavallee explained during an interview on Friday. Certainly, a commemoration for National Indigenous Veterans Day is something that were more than happy to support.

Lavallee said Indigenous people have played significant roles in all aspects of the Canadian Forces, citing Sgt. Tommy Prince of the Canadian Army as just one example. The RCAF, he explained, is always happy to honour that legacy.

They played an important role and made important contributions, and its important that we acknowledge that, he said. This is a way where we can do that, and were certainly happy to (offer) support.

The CF-18 will fly over the event at no lower than 500 ft above the highest obstacle on its route. The events are carefully planned, coordinated and controlled for public safety reasons, and can be cancelled due to bad weather or poor flying conditions.

Beardys Chief and Council have officially declared Nov. 8-11 as Veterans Week in the community. Events include a wreath laying ceremony and veterans fundraising dinner on Nov. 8, a veterans virtual round table discussion on Nov. 9, a virtual Remembrance Day ceremony on Nov. 10, and a Pipe Ceremony and in-person Remembrance Day ceremony on Nov. 11.

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CF-18 Hornet to fly over Beardy's and Okemasis in honour of Indigenous veterans - Prince Albert Daily Herald

INGELHEIM, Germany--(BUSINESS WIRE)--Boehringer Ingelheim, IP Group, the UK Cystic Fibrosis Gene Therapy Consortium (GTC, consisting of researchers from Imperial College London and the Universities of Oxford and Edinburgh) and Oxford Biomedica (OXB), announced today that Boehringer Ingelheim has exercised its options on intellectual property and know-how from the partners to progress and further accelerate the development of a potential, new treatment option for patients with CF. In the partnership, IP Group, acting on behalf of the three GTC host Universities, is granting exclusive global rights to develop, manufacture, register, and commercialize this lentiviral vector-based gene therapy for the treatment of cystic fibrosis. The GTC is additionally contributing its knowledge in pre-clinical research and clinical gene therapy development. OXB is adding its leading competence in manufacturing lentiviral vector-based therapies to Boehringer Ingelheims expertise in the development of novel breakthrough therapies for respiratory diseases.

CF is a rare, progressive, life-threatening disease that results in severe dysfunction and persistent infections of the lung affecting 70,000 people worldwide. It is caused by a defective or absent protein that results from mutations in the CFTR gene. This innovative development partnership among academia, life science investors, pharma, and biotech focusses on the advancement of BI 3720931, a novel, replication deficient lentiviral vector, in an inhaled formulation, which selectively introduces a healthy CFTR gene into the relevant target cells.

Professor Eric Alton, Coordinator of the GTC, said: The novel lung-targeting technology we have developed has demonstrated high gene transfer efficiency in pre-clinical models and offers the possibility of repeated administration to maintain a therapeutic effect, a benefit that other viral-based gene therapies may not be able to provide. Our novel therapy has the potential to improve CFTR function and modify disease in all CF patients, independent of the more than 2,000 different known gene mutations. The immediate target is those patients who are not eligible for CFTR modulators. The GTC is very excited to have reached this milestone after 21 years of focused effort. We are very grateful to our wonderful team and those with CF who have supported us in many ways including taking part in the multiple trials. We would like to thank our funders, both past and present, including the Health Innovation Challenge Fund (a partnership between Wellcome and the Department of Health and Social Care) and Just Gene Therapy, as well as the CF Trust, National Institute for Health Research and the Medical Research Council.

Since 2018, Boehringer Ingelheim has sponsored research and development activities with the GTC and OXB. The shared success achieved with our partners in this potentially revolutionary project makes us confident that we can now further accelerate this highly innovative therapeutic approach, said Clive R. Wood, Ph.D., Corporate Senior Vice President and Global Head of Discovery Research at Boehringer Ingelheim. With our leadership in the discovery and development of therapies in respiratory diseases combined with the gene therapy and manufacturing knowledge of our partners, we aim to bring the next breakthrough to patients suffering from CF, who are desperately waiting for better options.

John Dawson, Chief Executive Officer of Oxford Biomedica, said: We have enjoyed working with Boehringer Ingelheim, IP Group, and the GTC since 2018. Building on the great progress made to date, we are delighted that Boehringer Ingelheim, one of the worlds leading respiratory medicine organizations, has chosen to exercise the option to license OXB lentiviral vector manufacturing technology for this highly innovative inhaled cystic fibrosis gene therapy formulation developed by the GTC. This partnership is central to our companys mission of delivering life changing gene therapies to patients and has the potential to provide a new therapeutic option for many cystic fibrosis patients globally.

Under the terms of the option and license agreement with Boehringer Ingelheim, originally announced in August 2018, Boehringer Ingelheim will pay IP Group, on behalf of the GTC, an option exercise fee, near term, success based development, regulatory and sales milestone payments as well as royalties on net sales. OXB will receive an option exercise fee of 3.5 million and will be entitled to payments in an aggregate amount of up to 27.5 million upon achievement of various development, regulatory and sales milestones, in addition to a tiered low single digit royalty on net sales of a cystic fibrosis gene therapy product.

David Ramsden, Chief Executive of the Cystic Fibrosis Trust said: It is great news that Boehringer Ingelheim have committed to the next stage of the development of a gene therapy treatment for people with cystic fibrosis. This is an important step as it brings hope to the whole cystic fibrosis community and in particular to those who dont benefit from the currently available medicines. All of those who have helped us to invest long term in the work of the UK CF Gene Therapy Consortium should be proud of what they have made possible.

Please click on the following link for Notes to Editors:

http://www.boehringer-ingelheim.com/press-release/cystic-fibrosis-genetherapy-development-option-excercise

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Boehringer Ingelheim and Partners to Accelerate Development of First-In-Class Gene Therapy for Patients with Cystic Fibrosis - Business Wire

LOS ANGELES -- When the Dodgers acquired Trea Turner at the Trade Deadline on July 30, Gavin Lux walked into manager Dave Roberts office and offered to play the outfield. Lux had no previous experience in the outfield, having played his whole baseball career as either a shortstop or a second baseman, a skill set that made him one of the top prospects in baseball.

But Lux understood what the roster looked like and he wanted to find a way to get in the lineup. When he was sent down to Triple-A Oklahoma City at the end of August, Lux was understandably upset. Once he got to the Minors, he borrowed Zach Reks outfield glove and got to work. After just three games in the outfield at Triple-A, the Dodgers called him back up.

I think its a credit to him [being] willing to do whatever it takes to help us win, Roberts said before Wednesday's Game 4 of the National League Championship Series against the Braves. And its not easy to be in a position that you havent played a whole lot of, certainly in this stage, and on the heels of what happened yesterday. I mean, it takes a tough ballplayer to withstand that stuff.

As expected, Luxs adjustment to the outfield has come with its fair share of ups and downs. Even after Luxs costly misplay in Game 3, the Dodgers started him back in center field in Wednesday's 9-2 Game 4 loss. Batting in the five-hole, he finished 0-for-2 before being taken out for defense in the sixth.

Gavin is extremely talented and has been put in a position thats extremely difficult in playing a position hes never played before September and now in the playoffs, Dodgers right-hander Walker Buehler said. And in talking to him after [Game 3], I think hes going to learn a lot just on how to make that specific play. Gavins confident enough and talented enough to handle that kind of stuff.

That specific play came in the fourth inning when a ball bounced off his glove near the wall in right-center. It set up the Braves four-run inning and was ruled an error after a scoring change. The Dodgers came back to win Game 3, 6-5, with an eighth-inning rally.

It came in Luxs 10th career start at the position and also after Lux helped get the Dodgers out of a potential big frame earlier in the game when he caught an Ozzie Albies fly ball and threw to second base to retire Eddie Rosario for a double play. During that sequence, Lux showed off the instincts and speed that make the Dodgers comfortable with using the former top prospect in center field.

On Wednesday, Lux was involved in another critical play when Joc Pederson shot a flare into center field. Lux played the ball conservatively, and Pederson tallied an RBI single. Back on the mound, starter Julio Uras raised his arms up in frustration. The ball had a 40 percent catch probability, according to Baseball Savant. It also gave the Braves a four-run lead.

Changing positions this late into the season is indeed extremely difficult, but Lux has responded by becoming an integral part of the Dodgers lineup this October, going 3-for-14 with four walks. He wrapped up the regular season by hitting .360 after getting recalled from the Minors.

The outfield isnt necessarily the position for Lux moving forward, but it gives him the best chance to play. And the Dodgers are going to stick with him. Even if it doesnt always look natural.

I think hes a heck of a ballplayer, Roberts said.

When asked how many times hes watched Cody Bellingers tying homer in Game 3, Roberts said hes gone through it about a dozen times. The way he has relived the moment will probably surprise you.

Now with this thing called TikTok, its around everywhere, so my kids show me this stuff, Roberts said.

Wait, so does Roberts have his own TikTok?

No. No, he said with a smile. I might have a burner Twitter account, but I dont have a TikTok account.

Maybe thatll change if the Dodgers win the World Series.

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'Tough ballplayer': Lux learning CF on the job - MLB.com

NEW YORK The shifting nature of adaptation for Pseudomonas aeruginosa microbes found in the upper respiratory tract of cystic fibrosis patients with chronic rhinosinusitis may provide treatment clues that are not obvious from lung-centered microbial sampling, new research suggests.

"Our findings underscore the importance of infection-site biogeography to pathogen evolution and the relevance of the sinuses to overall CF respiratory health," co-senior and co-corresponding author Jennifer Bomberger, a microbiology and molecular genetics researcher at the University of Pittsburgh School of Medicine, and her colleagues wrote.

As they reported in a paper published in Cell Reports on Tuesday, the researchers used whole-genome sequencing, targeted 16S ribosomal RNA gene sequencing, "microbial identification after passive clarity technique" hybridization chain reaction (MiPACT-HCR)-based imaging, phenotyping, and other approaches to assess samples collected over time from half a dozen adult CF patients. Rather than analyzing lung sputum samples, they focused on less characterized P. aeruginosa communities in the upper respiratory tract, where early colonization occurs.

"Much of what we know about P. aeruginosa communities in cystic fibrosis is from sputum samples from the lungs," Bomberger explained in an email, noting that "our study is different in that we are analyzing P. aeruginosa evolution in the upper respiratory tract (i.e. sinuses)."

The team's results revealed distinct genomic features and adaptation events in the upper respiratory tract during different stages of infection and in relation to the size of the P. aeruginosa community considered.

Whereas early-stage infections involving large P. aeruginosa populations showed signs of selection related to the host environment and related treatments, leading to a rise in aggregation-prone bugs, later-stage infections tended to involve smaller, host-restricted population fragments that were prone to genome degradation and "mutator"-driven genetic drift effects.

"We propose that following initial adaptive evolution in larger populations under strong selection for aggregation, P. aeruginosa persists in small, fragmented populations that experience stronger effects of genetic drift," the authors reported.

The results hinted that MiPACT-HCR imaging or genome degradation signatures such as pseudogenization may help in distinguishing between P. aeruginosa populations in the process of selection and the genetic drift-prone populations found at later stages of the infection process.

Such differences in population size, structure, and evolutionary patterns may help in guiding therapeutic approaches, Bomberger noted, since P. aeruginosa populations with alterations that have arisen randomly through genetic drift are less likely to respond to targeted treatments, whereas populations under selection may be more amenable to therapeutic approaches targeting key mutations.

"This information would allow researchers and clinicians to know if the community is undergoing selection or more genetic drift and how likely mutations are to suggest selective pressures that could be effectively targeted therapeutically," she said.

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Cystic Fibrosis Sinus Infections Involve Microbes With Shifting Evolution Patterns - GenomeWeb

A unique interaction between CFTR the protein defective in people with cystic fibrosis (CF) and two pro-inflammatory proteins may explain why some CF patients develop liver disease, researchers reported.

Their work suggests that the CFTR protein can act as an anchor, bringing certain proteins including the pro-inflammatory beta-catenin and the p65 subunit of NF kappa B together to prevent their activation.

When CFTR is missing or dysfunctional, these proteins are activated, which can lead to the overgrowth of cells lining the bile ducts called biliary epithelial cells or BECs and cause liver inflammation. (Bile ducts are thin tubes that go from the liver to the small intestine.)

This missing interaction is at the core of the pro-inflammatory environment in the fibrotic liver it adds insult to injury and makes the disease worse, Satdarshan (Paul) Monga, MD, a professor of pathology and medicine at the University of Pittsburgh and the studys senior author, said in a university press release.

Findings were reported in the study -Catenin-1 NFB-CFTR interactions in cholangiocytes regulate inflammation and fibrosis during ductular reaction published in the journal eLife.

Why liver disease affects some CF patients is not well understood. A team led by researchers at the University of Pittsburgh investigated this, looking at how the expansion of biliary epithelial cells, an important repair process following liver injury or disease, is associated with inflammation and fibrosis.

Researchers created two different mouse models using genetic tools. The KO1 model had beta-catenin loss in both hepatocytes (the main functional liver cells) and in BECs (also known as cholangiocytes). The KO2 model had loss of beta-catenin only in hepatocytes.

Beta-catenin is well known to play a key role in hepatocyte proliferation, allowing for liver repair with injury or disease.

Mice in both models were exposed for two weeks to a special diet (choline-deficient, ethionine-supplemented diet) that induces liver injury. They were then allowed to recover on a normal diet, and assessed two weeks, three months, and six months later.

In both models, researchers saw a greater degree of cell death, inflammation, and fibrosis than was evident in wild-type (normal) mice. However, the two models seemed to show different recovery profiles.

Two weeks after switching to normal diet, KO1 mice continued to show greater injury due to problems with hepatocyte proliferation. Even six months later, KO1 mice had evidence of progressive ductular reaction a proliferation of reactive bile ducts induced by liver injury which is associated with inflammation and fibrosis. This suggested a morphology close to that seen in CF patients.

Analysis of beta-catenin levels and the expression (activity) of Ctnnb1, the gene which encodes beta-catenin, showed an absence of beta-catenin at the six-month assessment in KO1 mice. In contrast, in wild-type mice also given the injury-inducing diet, beta-catenin was detected and fibrosis seemed to resolve after two weeks on a normal diet.

Better repair with time was also evident in KO2 mice after moving to a regular diet. By six months, fibrosis seemed to have resolved in this model and Ctnnb1expression was comparable to that of wild-type mice. Ductular reaction had also resolved in KO2 mice at six months, although not at three months.

We observed highly divergent injury-repair responses in the two models. KO2 mice showed progressive repair through expansion of BEC-derived beta-catenin-positive hepatocytes, and resolution of inflammation, [ductular reaction] and fibrosis, the researchers wrote.

Beta-catenins lack in the BECs of the KO1 model was thought to cause sustained activation of the NF kappa B protein a crucial regulator of immune and inflammatory responses upon injury, leading to greater inflammation. NF kappa B is activated through the interaction of the beta-catenin protein with p65 (a subunit of NF kappa B).

In contrast, in KO2 mice that lacked hepatocytes but not beta-catenin, thebeta-cateninp65 complex was able to be re-established, thus preventing chronic [NF kappa B] activation, the researchers wrote.

KO2 show gradual liver repopulation with BEC-derived b-catenin-positive hepatocytes, and resolution of injury, they noted, which mice in the KO1 model did not.

Researchers next worked with liver tissue samples from multiple sources, including CF patients, to determine if the interactions seen in mouse models and cell studies could be replicated. Specifically, they were looking for how p65 and beta-catenin may be interacting with the CFTR protein.

Turning off, or stopping, expression of the CFTR gene so that no CFTR protein is produced led to a pronounced activation of p65, the researchers found. Liver tissue from a CF patient was also seen to have lower-than-usual levels of total beta-catenin.

Doing an experiment and confirming for the first time that what we saw in mice was also what we see in patients with cystic fibrosis was very exciting and validating, said Shikai Hu, the studys first author.

Based on these results, the team suggested that the CFTR protein acts as a molecular anchor, interacting with beta-catenin-1 and p65 to prevent their activation. When CFTR is absent or defective, as in CF, these proteins become activated and ultimately induce progressive liver inflammation and fibrosis.

We report a novel beta-catenin-NF kappa B-CFTR interactome in BECs, and its disruption may contribute to hepatic pathology [liver disease] of CF, the researchers concluded.

More studies are need, they added, to further understand the molecular changes caused by CF in the liver.

Excerpt from:

Liver Disease in CF Appears Linked to Problems in CFTR Protein - Cystic Fibrosis News Today

A Michigan State University College of Human Medicine researcher has been awarded a federal grant to study a gene therapy treatment for cystic fibrosis, a potential cure for the rare, lethal disease that afflicts more than 30,000 people in the United States and 70,000 worldwide.

Dr. Xiaopeng Li

The grant from the National Institutes of Health will allow a team of researchers led by Xiaopeng Li, PhD, an associate professor in the Department of Pediatrics and Human Development, to study how CFTR gene (the causal gene for cystic fibrosis) mutations in the small airways in the lungs of cystic fibrosis patients lead to life-threatening infections.

The funding $2.1 million over four years is critical for this research, Li said. One reason we got the grant is we have a lot of data to show this is a feasible approach to treating the disease.

Li will lead a team that includes College of Human Medicine researchers Jeremy Prokop and Christopher Waters. The team also includes four pulmonologists Reda Girgis, Susan Millard and John Schuen from Spectrum Health and Helen DeVos Childrens Hospital Cystic Fibrosis Center, and Ryan Thomas from MSU's College of Human Medicine.

Marrying basic science and clinical medicine is a goal that is driving the collaboration between MSU and Helen DeVos Childrens Hospital/ Spectrum Health, said John Schuen, MD, chair, Cystic Fibrosis Translational Research Program. This relationship has already led to significant grant opportunities and published literature. Together, we will help uncover important discoveries at the cellular level that we hope will benefit our CF patients in the future.

The Cystic Fibrosis Translational Research Program is sponsored by the MSU-Spectrum Health Alliance and the Hunt for a Cure Foundation. The collaboration between MSU and Spectrum Health highlights the importance of our outstanding and expanding research partnership in the area of cystic fibrosis, said B. Keith English, MD, chair of the colleges Department of Pediatrics and Human Development.

The significance of the small airways in cystic fibrosis patients has not been well studied, Li said. Previous research, however, has shown that cells in the small airways of cystic fibrosis patients do not secrete bicarbonate, a substance necessary to maintain a proper pH balance on the cell surface. As a result, Li said, cystic fibrosis patients tend

to develop thick mucus obstruction in the small airways, making them susceptible to bacterial infections, which can lead to respiratory failure and death.

For years, patients have had to spend hours using drug therapies and airway clearance. More recently, CFTR modulators have been approved by the FDA but still do not provide a cure, with life expectancy in the mid-40s. It is a genetic disease with over 2,000 mutations in the CFTR gene. CF is usually diagnosed in infancy but even adults can discover that their chronic cough or recurrent pancreatitis is actually CF.

The study will look at why the cystic fibrosis small airways are so vulnerable to infection and the feasibility of replacing the defective gene with a normal gene, a procedure that would be tantamount to a cure Li said.

Its a particularly devastating disease, he said. Hopefully, one day we can apply this treatment to human patients.

Research reported in this publication was supported by the National Heart, Lung, and Blood Institute of the National Institutes of Health under Award Number R01HL153165. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

Read more:

MSU, Spectrum Health partner on over $2M NIH gene therapy study for cystic fibrosis - MSUToday

- 96-week interim results of TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) study show no loss of pulmonary function in people with at least one F508del allele, a first for any CFTR modulator

- Real-world data from people treated with KALYDECO (ivacaftor) over approximately 6 years show lower rates of mortality, lung transplant and pulmonary exacerbations than comparator cohort -

- Additional presentations highlight safety and efficacy profile of TRIKAFTA -

BOSTON, October 19, 2021--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that five scientific abstracts about the companys portfolio of cystic fibrosis (CF) medicines will be presented at the 2021 North American Cystic Fibrosis Conference (NACFC) taking place virtually November 2-5, 2021.

Key data being presented include 96-week interim results from an ongoing TRIKAFTA open-label extension study in people with CF ages 12 years and older with F508del/Minimal Function (F/MF) or F508del/F508del (F/F) genotypes, showing that the favorable safety profile and clinically meaningful improvements in lung function, respiratory symptoms and CFTR function as measured by sweat chloride observed in the Phase 3 pivotal studies were maintained through an additional 96 weeks of treatment (Poster #681). Additionally, a post hoc analysis of the annualized mean rate of change in percent predicted forced expiratory volume in 1 second (ppFEV1) showed there was no loss of pulmonary function over 96 weeks in this CF population, which is a first for any CFTR modulator to date.

Also presented at this years conference are data on results from a retrospective study of patients with gating mutations ages 6 years or older treated with KALYDECO showing that people treated with KALYDECO over approximately six years of follow up had significantly lower rates of mortality, lung transplant and pulmonary exacerbations (PEx) compared to a cohort of patients that were not eligible for and not receiving KALYDECO treatment (Poster #178).

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"The data were presenting this year clearly demonstrate that our portfolio of CFTR modulators has truly transformed the CF treatment landscape," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "The long-term follow up data from TRIKAFTA in particular demonstrates the unprecedented treatment effect of this medicine and reinforces the high bar it sets for safety and efficacy. Were committed to continuing to serially innovate in our CF program until we reach our goal of bringing transformative medicines to everyone with this disease."

Additional Presentations

In addition to the studies noted above, other presentations at NACFC include:

INTERIM RESULTS FROM THE HELIO STUDY: Interim analysis of a study of the real-world clinical effectiveness of TRIKAFTA in people with CF age 12 years and older with at least one F508del allele who were ineligible for another CFTR modulator, demonstrating clinically meaningful improvements in lung function and nutritional status at 6 months. In addition, the annualized PEx rate was lower with TRIKAFTA treatment. These results are consistent with findings from pivotal clinical trials (Poster #56).

QUALITATIVE STUDY OF PATIENTS TREATED WITH TRIKAFTA AND CAREGIVERS: Results from an ongoing qualitative study to evaluate (1) the real-world patient experience of TRIKAFTA treatment from the perspective of people with CF and caregivers and (2) the impact of TRIKAFTA on the caregiver experience. The impact of the SARS-CoV-2 pandemic was also included in the assessment of the patient and caregiver experience. Results from this study demonstrate that TRIKAFTA has a meaningful and substantial impact on the daily lives of people with CF and caregivers, including the ability to cope with living through the SARS-CoV-2 pandemic (Poster #285).

INTERIM RESULTS FROM A PHASE 3 OPEN-LABEL EXTENSION STUDY OF CHILDREN WITH CF AGES 6 YEARS AND OLDER: An interim analysis at week 24 of an ongoing, 96-week, Phase 3, open-label extension study designed to assess the long-term safety and efficacy of TRIKAFTA in children 6 years of age and older with at least one F508del allele. Results were consistent with the previously established safety profile of TRIKAFTA in this age group. Results also showed robust and clinically meaningful improvements in lung function, respiratory symptoms, and CFTR activity as measured by sweat chloride and indicate TRIKAFTA provides long-term benefit in this younger patient population (Poster #562).

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes one from each parent to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.

About KALYDECO (ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein at the cell surface does not function properly. Known as a CFTR potentiator, ivacaftor is an oral medicine designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. KALYDECO (ivacaftor) was the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO (ivacaftor)

KALYDECO (ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 4 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 4 months of age.

Patients should not take KALYDECO if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. Johns wort.

Before taking KALYDECO, patients should tell their doctor if they: have liver or kidney problems; drink grapefruit juice or eat grapefruit; are pregnant or plan to become pregnant because it is not known if KALYDECO will harm an unborn baby; and are breastfeeding or planning to breastfeed because is not known if KALYDECO passes into breast milk.

KALYDECO may affect the way other medicines work, and other medicines may affect how KALYDECO works. Therefore the dose of KALYDECO may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.

KALYDECO can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO affects them.

Patients should avoid food containing grapefruit while taking KALYDECO.

KALYDECO can cause serious side effects.

High liver enzymes in the blood have been reported in patients receiving KALYDECO. The patients doctor will do blood tests to check their liver before starting KALYDECO, every 3 months during the first year of taking KALYDECO, and every year while taking KALYDECO. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.

Abnormality of the eye lens (cataract) has been noted in some children and adolescents receiving KALYDECO. The patients doctor should perform eye examinations prior to and during treatment with KALYDECO to look for cataracts.

The most common side effects include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.

These are not all the possible side effects of KALYDECO. Please click here to see the full Prescribing Information for KALYDECO.

About TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.

INDICATIONS AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA

TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 6 years of age.

Patients should not take TRIKAFTA if they take certain medicines or herbal supplements, such as: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. Johns wort.

Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.

TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. Therefore, the dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should especially tell their doctor if they take: antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin.

TRIKAFTA may cause dizziness in some people who take it. Patients should not drive a car, operate machinery, or do anything that requires alertness until they know how TRIKAFTA affects them.

Patients should avoid food or drink that contains grapefruit while they are taking TRIKAFTA.

TRIKAFTA can cause serious side effects, including:

Liver damage and worsening of liver function in people with severe liver disease that can be serious and may require transplantation. Liver damage has also happened in people without liver disease.

High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patients doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.

Abnormality of the eye lens (cataract) has happened in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.

The most common side effects of TRIKAFTA include headache, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, diarrhea, rash, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, flu (influenza), inflamed sinuses, and increase in blood bilirubin.

These are not all the possible side effects of TRIKAFTA. Please click here to see the full Prescribing Information for TRIKAFTA.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit http://www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Carmen Bozic in this press release, statements regarding the potential benefits, safety and efficacy of TRIKAFTA and KALYDECO, and our plans to present data about our portfolio of CF medicines at the NACFC, including data from our TRIKAFA open-label extension study, a retrospective study of patients treated with KALYDECO, and additional scientific presentations regarding TRIKAFTA. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under the heading "Risk Factors" in Vertex's most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company's website at http://www.vrtx.com and on the SECs website at http://www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

View source version on businesswire.com: https://www.businesswire.com/news/home/20211019005260/en/

Contacts

Vertex Pharmaceuticals Incorporated Investors: Michael Partridge, +1 617-341-6108orBrenda Eustace, +1 617-341-6187orManisha Pai, +1 617-429-6891

Media: mediainfo@vrtx.com orU.S.: +1 617-341-6992orHeather Nichols: +1 617-839-3607orInternational: +44 20 3204 5275

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Vertex to Present Long-Term Data Demonstrating Significant Benefits of Treatment With CFTR Modulators at North American Cystic Fibrosis Conference...