Trikafta has transformed my life in ways I never thought possible. But with that came a sharp awareness of those still waiting for a treatment that works for them. That’s why I’m deeply committed to ...

Human Rights Watch welcomes the opportunity to provide input to the Committee on the Rights of Persons with Disabilities (the “Committee”) in advance of its upcoming public session on the situation of ...

Human Rights Watch welcomes the opportunity to provide input to the Committee on the Rights of Persons with Disabilities (the "Committee") in advance ...

Sionna has closed its IPO, raising $191m for cystic fibrosis candidates that it thinks could be more effective than rivals from Vertex ...

Vertex’s quest to treat a wider group of patients with cystic fibrosis (CF) continues, after the FDA approved its latest combination therapy.

Liver fibrosis is the liver's response to injury, in which excess extracellular matrix is deposited. In most cases, liver fibrosis is the result of chronic liver disease, such as hepatitis.

Olivia had been showing symptoms since she was just 7 months old — including a belly that remained swollen, hard to the touch and rounder than it should have been. Her pediatrician brushed it off as a ...