Nature

Congenital Bilateral Absence of Vas Deferens and CFTR Gene Mutations

Congenital bilateral absence of the vas deferens (CBAVD) is a recognised cause of male infertility most frequently linked to mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) ...

Vertex Pharma wins FDA label expansion for cystic fibrosis therapies

Vertex Pharmaceuticals (VRTX) stock is in focus as the FDA greenlights label expansions for its cystic fibrosis drugs, ...
Click2Houston

Genetic medicine can leave people with rare mutations behind. But there's new hope

This photo provided by Emilys Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare genetic mutation, during a trip to Maine. (Emilys Entourage via AP) ...
Nasdaq

Vertex Announces UK MHRA Approval of ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor), a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis

- Deutivacaftor/tezacaftor/vanzacaftor approved for people with cystic fibrosis 6 years and older with at least one responsive mutation in the CFTR gene, including ...
Labroots

A Gene Editor to Correct a Common Cystic Fibrosis Mutation

Cystic fibrosis (CF) is a common genetic disorders that has been well studied. Researchers have identified CF-causing mutations in a gene called CFTR, which encodes for an ion channel. The genetic ...
Nasdaq

Vertex Announces US FDA Approval of ALYFTREK™, a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies - - In head-to-head ...