All articles tagged with #cystic fibrosis
Originally Published 3 days ago — by Krystal Biotech, Inc
Krystal Biotech announced positive interim results from its Phase 1 trial of KB407, a gene therapy for cystic fibrosis, confirming successful lung delivery and expression of wild-type CFTR protein across diverse patients, paving the way for a larger, repeat-dosing study expected to start in 2026.
Originally Published 4 months ago — by statnews.com
Three scientists, including researchers from Vertex Pharmaceuticals and the University of Iowa, received the prestigious Lasker Award for their groundbreaking work that significantly improved cystic fibrosis treatment and extended patient lifespans.
Originally Published 4 months ago — by Live Science
Three scientists received the $250,000 Lasker-DeBakey Award for developing Trikafta, a groundbreaking therapy that significantly extends the lifespan of cystic fibrosis patients by targeting the disease's genetic root, leading to improved quality of life and survival rates.
Originally Published 4 months ago — by The Boston Globe
Three scientists from Vertex Pharmaceuticals and the University of Iowa received the prestigious Lasker Award for their groundbreaking work that significantly improved treatment and extended the lives of cystic fibrosis patients.
Originally Published 5 months ago — by BBC
Yvonne Hughes, diagnosed with cystic fibrosis as a baby and once planning her funeral, has experienced a life-changing breakthrough with the drug Kaftrio, allowing her to live beyond her mid-40s and perform comedy at the Edinburgh Fringe, turning her health struggles into inspiring material.
Originally Published 1 year ago — by The New York Times
Despite universal newborn screenings for cystic fibrosis, children of color often face delays in diagnosis due to medical biases and misconceptions that the disease primarily affects white people. Rena Barrow-Wells' experience with her sons highlights the ongoing struggle for timely and accurate diagnosis in minority communities.
Originally Published 1 year ago — by The Guardian
South African patients with cystic fibrosis are taking legal action against Vertex Pharmaceuticals for access to the life-changing drug Trikafta, which costs $326,000 a year per patient. The lawsuit accuses the company of patent abuse and violating patients' human rights, as the drug is unaffordable for most. While Vertex defends its intellectual property rights, patients are resorting to importing a generic version from Argentina at a fraction of the cost. The legal battle highlights the challenges of accessing expensive medication in low- and middle-income countries, leaving many patients without life-saving treatment.
Originally Published 1 year ago — by Longreads
The introduction of the drug Trikafta has given cystic fibrosis patients a chance at a longer life, prompting them to navigate a future they never expected. Jenny, a patient, found herself redefining her purpose and pursuing a career in social work after realizing the uncertainty of no longer being actively dying. She now advocates for mental health and is pursuing a master's degree, finding fulfillment in helping hospice patients navigate the end of life with dignity and small acts of kindness.
Originally Published 1 year ago — by Yahoo Entertainment
Taylor Stephenson, a 26-year-old woman with cystic fibrosis, recently underwent her third double lung transplant in eight years at Duke Health after her second set of lungs began to fail. Following the successful surgery in January, she has made significant progress in her recovery, including walking 200 ft. just a day after waking up from the surgery. Stephenson hopes her story will inspire others with cystic fibrosis to consider a third transplant as a viable option and to not give up hope.
Originally Published 1 year ago — by New York Post
Taylor Stephenson, a 26-year-old cystic fibrosis patient, successfully received her third set of lungs after her second transplant failed. Despite facing rejection and complications, she underwent a successful third transplant at Duke University Hospital. Now at 50% lung capacity, she aims to climb three mountains and hopes to be an advocate for others going through similar struggles, emphasizing the message to "keep fighting."
Originally Published 2 years ago — by The Guardian
The National Institute for Health and Care Excellence (Nice) has published draft guidance stating that Orkambi and Kaftrio, gene-therapy drugs that have significantly improved the prognosis of people with cystic fibrosis (CF), are too expensive and are no longer recommended for use on the NHS. The cost of a month's supply of Kaftrio is £8,346.30 before VAT. If Nice upholds its recommendation, children under six years old and those born after them will not be able to access the drugs, potentially leaving their future uncertain. The author calls for an alternative solution, such as issuing a compulsory Crown use license to secure an affordable, generic version of the drugs for NHS patients.
Originally Published 2 years ago — by BBC.com
Georgia Burchell, the mother of a 15-month-old baby with cystic fibrosis, is anxiously waiting for the NHS to decide whether to fund drugs that could double her daughter's life expectancy. The drugs, Orkambi, Symkevi, and Kaftrio, are considered "lifesaving" and could provide a normal life for her daughter. However, the cost of treatment, which exceeds £100,000 per patient per year, may make it unaffordable for the NHS. While her daughter is due to start Orkambi soon, she cannot begin Kaftrio until she is two years old. The final decision is expected to be announced next year.
Originally Published 2 years ago — by Yahoo Finance
Vertex Pharmaceuticals missed sales estimates for its older cystic fibrosis (CF) treatments in the third quarter, with a 35.4% decline in sales. The company now expects annual sales of approximately $9.85 billion from its CF treatments. However, sales of its top-selling CF drug, Trikafta, came in at $2.27 billion, slightly exceeding estimates. Vertex anticipates continued growth in the number of CF patients taking its medicines, including through new approvals and reimbursement for treatment of younger patients.
Originally Published 2 years ago — by Investor's Business Daily
Vertex Pharmaceuticals' sales of its cystic fibrosis treatment, Trikafta, missed some estimates, causing the company's stock to tumble. Revenue from Trikafta during the September quarter was $2.27 billion, slightly below the estimate of $2.33 billion. Additionally, other cystic fibrosis drugs brought in $209.2 million in sales, missing forecasts. Vertex also scrapped the development of a potential liver disease treatment and needs further testing for a gene-editing treatment for Duchenne muscular dystrophy. Despite the mixed sales report, Vertex raised its outlook for the year, expecting its cystic fibrosis treatments to generate $9.85 billion in sales.
Originally Published 2 years ago — by Yahoo Finance
Vertex Pharmaceuticals reported its financial results for the third quarter of 2023, with product revenue increasing by 6% to $2.48 billion compared to the same period last year. The company raised its full-year 2023 product revenue guidance to approximately $9.85 billion. Vertex's R&D pipeline continues to advance, with Phase 3 data in cystic fibrosis and acute pain expected in early 2024, and Phase 2 data in neuropathic pain expected in late 2023. The company is also preparing for potential new product launches, including exa-cel in sickle cell disease and transfusion-dependent beta thalassemia.