All articles tagged with #cystic fibrosis
Elizabeth Wehrle, a cystic fibrosis patient and former lung transplant recipient, received a two-stage quadruple-organ transplant at Northwestern Memorial Hospital—lungs, liver, and kidney—from one donor after severe rejection; the groundbreaking surgery demonstrates complex coordination and offers hope for similar patients.
Lisa Zaccaria recounts growing up with cystic fibrosis alongside her sister Michelle, whose death at 35 jolted her out of fear, and how discovering Linda Ellis’s poem 'The Dash' spurred her to live boldly—parasailing in Key Largo and taking risks she’d once avoided—to honor Michelle and live a life without regret.
Jasmine Jones, 28, underwent a groundbreaking 36-hour quadruple transplant at University of Chicago Medicine in January, receiving a new right and left lung, kidney, and liver from one donor—the first known procedure of its kind in Illinois. Five months later she’s back home and doing well, with doctors saying the multi-organ transplant shows how advances in perfusion and surgical technique are expanding options for patients with end-stage organ disease.
The Ethicist argues that using IVF with preimplantation genetic testing to avoid CF‑risk embryos can be ethically permissible, since selecting embryos is about health states rather than devaluing existing people. While the term “eugenics” is loaded, the key is whether such selection expresses contempt for those with the condition. Personal motives (like wanting a daughter) should be weighed carefully, and the analysis acknowledges the limits of genetic testing and the importance of valuing the current child while considering future risks.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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."
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.