By the time Enid Katz was two years old, she had already suffered through several bouts of bronchitis and was always hungry, ...

Vertex Pharmaceuticals (NASDAQ:VRTX), a global biotechnology company with a market capitalization of $119.1 billion known for its transformative cystic fibrosis (CF) treatments, finds itself at a ...

A Wicklow mother is among a concerned group of parents who are calling for individual trials of the modulator therapies for ...

Vertex Pharmaceuticals (NASDAQ:VRTX), a leading biotechnology company known for its cystic fibrosis (CF) treatments, is at a critical juncture as it seeks to diversify its portfolio and maintain its ...

Allison John and Rocky When Allison John was 13 and watching the science programme Tomorrow’s World , a segment on gene therapy came up. This was cutting edge stuff in the early 1990s and John had ...

for the treatment of cystic fibrosis in patients with at least one F508del mutation or another treatment-responsive mutation in the CFTR gene. The fixed-dose triple-combination therapy ...

His rare gene means he is currently excluded from modulator therapy Kaftrio ... are being supported by Cystic Fibrosis Ireland, and believes the only solution for people with CF like Aaron ...

Boehringer Ingelheim (BI) and the UK Cystic Fibrosis Gene Therapy Consortium announced a global collaboration to develop a first-in-class, long-term therapy for patients with cystic fibrosis (CF).

Adoption of Triple Combination Therapy Accelerates at Unprecedented Rates as Precision Medicine Revolution ContinuesRockville, MD, Jan. 29, 2025 (GLOBE NEWSWIRE) -- According to Fact.MR, a market ...

The bottom line is few companies can match its combination of established leadership in rare diseases and realistic pathways ...

Vertex Pharmaceuticals (NASDAQ: VRTX) just snagged its second U.S. Food and Drug Administration (FDA) approval in only 41 ...