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Cystic Fibrosis

All articles tagged with #cystic fibrosis

Cystic Fibrosis’s Final 10% Face Treatment Gap as New Therapies Stall
health-and-science4 days ago

Cystic Fibrosis’s Final 10% Face Treatment Gap as New Therapies Stall

While 90% of cystic fibrosis (CF) patients benefit from Vertex Pharmaceuticals’ drugs Trikafta and Alyftrek, approximately 10% lack the specific genetic mutations required for these treatments to work. Emily’s Entourage, a nonprofit founded by patient Emily Kramer-Golinkoff, is funding research into alternative therapies, including gene treatments and antimicrobials, to address this gap. Recent setbacks, including halted trials by Vertex/Moderna and Boehringer Ingelheim, have delayed progress, but new approaches like Spirovant Sciences’ gene therapy and RNA-based methods offer hope. The foundation has raised over $22 million to support 51 projects, aiming to prevent the misconception that CF is fully cured and to accelerate treatments for rare genetic conditions.

via CNBC|
#clinical-trials#cystic-fibrosis#gene-therapy
RNA-tRNA therapy aims to bypass stop signals to treat multiple genetic diseases
health1 month ago

RNA-tRNA therapy aims to bypass stop signals to treat multiple genetic diseases

Scientists at the University of Toronto engineered suppressor tRNAs delivered in lipid nanoparticles to bypass premature stop signals, restoring full-length proteins in cystic fibrosis models. A specific chemical modification (m1A) boosted tRNA activity and persistence, while a lung-targeted delivery system (TTP-3) improved tissue reach. Because stop signals occur across many genes, this RNA-based approach could become a disease-agnostic platform, potentially treating numerous genetic disorders, though clinical safety and organ-specific delivery remain key hurdles before use in people.

via thebrighterside.news|
#cystic-fibrosis#genetic-diseases#health
Inhalable tRNA Therapy Reverses CF Defect, Paving Way for Other Genetic Diseases
science1 month ago

Inhalable tRNA Therapy Reverses CF Defect, Paving Way for Other Genetic Diseases

University of Toronto researchers engineered an inhalable, modified tRNA that can read through premature stop codons in CFTR mRNA, restoring CFTR protein in mice and persisting in lung tissue for up to 40 days; in patient-derived organoids, tRNA plus the CF drug Trikafta restored function where the drug alone failed. The study suggests tRNA therapies may complement mRNA approaches and could be extended to Duchenne muscular dystrophy and Rett syndrome, with plans to move toward clinical trials in 2–3 years.

via Fierce Biotech|
#cystic-fibrosis#gene-therapy#inhalable-rna
Quadruple organ transplant gives CF patient a new lease on life
health4 months ago

Quadruple organ transplant gives CF patient a new lease on life

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.

via Chicago Tribune|
#cystic-fibrosis#health#liver-transplant
Ethics of embryo selection: balancing CF risk, parental love, and eugenics concerns
ethics8 months ago

Ethics of embryo selection: balancing CF risk, parental love, and eugenics concerns

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.

via The New York Times|
#cystic-fibrosis#embryo-selection#ethics
"South Africans Fight for Access to Life-Saving Cystic Fibrosis Drug"
health2 years ago

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

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.

via The Guardian|
#cystic-fibrosis#health#patent-abuse
health-and-medicine2 years ago

"Unveiling the Aftermath of the Miracle Drug"

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.

via Longreads|
#cystic-fibrosis#health-and-medicine#hospice-care