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Trikafta

All articles tagged with #trikafta

Scientists Honored with Lasker Award for Cystic Fibrosis Breakthroughs

Originally Published 4 months ago — by Live Science

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Source: 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.

"South Africans Fight for Access to Life-Saving Cystic Fibrosis Drug"

Originally Published 1 year ago — by The Guardian

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Source: 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.

"Unveiling the Aftermath of the Miracle Drug"

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.

Vertex Pharma's Sales Fall Short Due to Lackluster Demand for Established CF Therapies

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.

Vertex Pharma's Q3 Earnings Fall Short Amid Weak Demand for CF Treatments

Originally Published 2 years ago — by Investor's Business Daily

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Source: 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.

"Joy and Challenges: The Impact of Vertex's Cystic Fibrosis Drug on Patients"

Originally Published 2 years ago — by The Boston Globe

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Source: The Boston Globe

Cystic fibrosis (CF) patients have long struggled with the debilitating effects of the genetic illness, which impairs breathing and digestion. However, recent advancements in CF research have brought hope to patients like Jessie Ulan. The development of the drug Trikafta, a protein-repairing medicine, has significantly improved the lives of CF patients by addressing the underlying cause of the disease. Trikafta has allowed patients to breathe easier, reduced the need for daily therapy and medications, and improved their overall quality of life. While challenges remain, the progress in CF treatment has brought joy and optimism to patients and their families.

Breakthrough Prize honors life-changing cystic fibrosis treatment and cancer pioneer with $3 million awards

Originally Published 2 years ago — by Nature.com

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Source: Nature.com

The chemists behind the development of Trikafta, a triple-drug combination that has significantly improved the lives of people with cystic fibrosis, have been awarded the prestigious Breakthrough Prize. Sabine Hadida, Paul Negulescu, and Fredrick Van Goor at Vertex Pharmaceuticals developed the treatment by combining different drugs that help a faulty protein to function. Trikafta has been hailed as one of the most remarkable achievements in biomedical research in the last 30 years. The Breakthrough Prize, which awards achievements in life sciences, physics, and mathematics, also recognized breakthroughs in Parkinson's disease research, CAR-T-cell immunotherapy for leukemia, and the study of conformal field theories in physics.

New Drug Saves Woman's Life, But With One Major Condition.

Originally Published 2 years ago — by HuffPost

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Source: HuffPost

Trikafta, the most powerful drug for cystic fibrosis, has been a game-changer for patients like Sammie, who was diagnosed with the disease at two years old. The drug targets the underlying defect of the disease and has been shown to increase survival rates and improve quality of life. However, not all patients can take the drug due to side effects or barriers to access, including high costs and disparities in insurance coverage. The author, whose daughter is on Trikafta, advocates for continued research, health insurance protections, and sharing the stories of those affected by the disease.