Medscape

FDA Expands Options for Cystic Fibrosis Patients

The FDA has expanded indications for two targeted combination treatments, Alyftrek and Trikafta, that will now benefit more ...

Vertex Pharma wins FDA label expansion for cystic fibrosis therapies

Vertex Pharmaceuticals (VRTX) announced on Wednesday that the U.S. FDA approved label expansions for Alyftrek and Trikafta, its treatments for cystic fibrosis, a hereditary disease caused by mutations ...
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) ...
Seeking Alpha

Vertex Announces European Commission Approval of ALYFTREK®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis

- ALYFTREK ® (deutivacaftor/tezacaftor/vanzacaftor) approved in the EU for people with CF 6 years and older with at least one non-class I mutation in the CFTR gene ...
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 ...
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) ...
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 ...