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 ...
Seeking Alpha

European Commission Approves Expanded Label for KAFTRIO® in Combination With Ivacaftor for People With Cystic Fibrosis

-Approximately 4,000 people living with CF in the European Union are newly eligible for a medicine that treats the underlying cause of their disease for the first time- “We have been working for years ...
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) ...
Los Angeles Times

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

Emily Kramer-Golinkoff can’t get enough oxygen with each breath. Advanced cystic fibrosis makes even simple things like walking or showering arduous and exhausting. She has the most common fatal ...