University of California7h
Breathing new life: Working toward an inhalable gene therapy for cystic fibrosis
Although cystic fibrosis is a single gene mutation, there are more than 1,000 different ways the CFTR gene can mutate in ...
Cystic Fibrosis Clinical Trials and Studies: EMA, PDMA, FDA Approvals, Mechanism of Action, ROA, NDA, IND, and Companies
In March 2025, Vertex Pharmaceuticals Incorporated announced a study is to evaluate the safety, and tolerability and efficacy ...
NHS hope as new miracle drug greatly reduces some cystic fibrosis symptoms
The genetic condition cystic fibrosis had no treatments on the NHS before 2019 and a Daily Express campaign - now a miracle ...
News-Medical.Net on MSN7d
CRISPR technologies paving the way for advances in regenerative medicine
A recent review published in the journal Engineering delves into the significant advancements and potential of CRISPR ...
McGill University Health Centre6d
Major funding for research project on cystic fibrosis
A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...
Mirage News13d
Triple Combo Drug Approved for Cystic Fibrosis
In two randomised phase 3 studies involving 480 participants aged 12 years and over, ivacaftor/tezacaftor/vanzacaftor was ...
PMLive9d
Vertex’s triple combination cystic fibrosis therapy Alyftrek approved by MHRA
Vertex Pharmaceuticals’ triple combination cystic fibrosis (CF) treatment has been approved by the Medicines and Healthcare ...
KATC2d
Cystic Fibrosis Foundation to recognize Acadiana’s top young professionals
The Cystic Fibrosis Foundation will host Acadiana’s Finest, a campaign to raise funds and awareness for cystic fibrosis and recognize the area’s top young professional leaders.
Where Will Vertex Pharmaceuticals Be in 10 Years?
The past decade has been a game-changing one for Vertex Pharmaceuticals (NASDAQ: VRTX). The biotech won approval for its ...
Express Healthcare3d
Rare disease treatment: Look at emerging therapies and medications
Dr Anup Rawool, Consultant in Clinical and Cancer Genetics at Sahyadri Hospitals, Pune, discusses innovative therapies and ...
The American Journal of Managed Care3d
Solving the Transition Conundrum as More Children With Muscular Dystrophy Live to Adulthood
Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...