SAN DIEGO--(BUSINESS WIRE)--Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global messenger RNA medicines company focused on the development of infectious disease ...
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) ...
Cystic fibrosis is a genetic disorder that affects roughly 40,000 Americans. The condition is caused by a mutation in the patient's cystic fibrosis transmembrane conductance regulator (CFTR) gene, ...
Could next-generation gene editing mean a cure for cystic fibrosis (CF)? There's been a lot of advancement on the disease in the last decade, but there are still those living with the disease who get ...
MENLO PARK, Calif.--(BUSINESS WIRE)--ReCode Therapeutics, a clinical-stage genetic medicines company using tissue-specific delivery to power the next wave of mRNA and gene correction therapeutics, ...
A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient transduction of airway epithelial cells to achieve therapeutic correction. Gene editing is a method to permanently ...
- 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 ...
States screen for cystic fibrosis (CF) but with significant variation among their screening algorithms. The Cystic Fibrosis Foundation group conducted a systematic review, leading to seven ...
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 ...
This photo provided by Emily’s Entourage in April 2025 shows Emily Kramer-Golinkoff, who has cystic fibrosis caused by a rare genetic mutation, during a trip to Maine. (Emily’s Entourage via AP) Emily ...