The Massachusetts-based biotech plans to use the funds to push its candidates into mid-stage clinical trials in a space ...

By the time Enid Katz was two years old, she had already suffered through several bouts of bronchitis and was always hungry, ...

Lately, one of America's leading pharmaceutical companies can't catch a break from Wall Street. Shares of Vertex ...

In December, the FDA approved Alyftrek, a new once-daily pill for patients with cystic fibrosis caused by at least one of 303 possible mutations in their CFTR genes. Alyftrek's precursor ...

In people with CF, mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause the CFTR protein to become dysfunctional. When the protein is not working correctly, it’s ...

Cystic Fibrosis ... 1 Mechanism of action and CFTR variants approved by the US Food and Drug Administrations and the Brazilian National Health Surveillance Agency. Modulators Action Mechanism FDA ...

Sionna Therapeutics is eyeing a $156 million IPO as the cystic-fibrosis-focused biotech looks to take its lead candidate into ...

Researchers have been trying for decades to stop the cells that damage the lung in people with pulmonary fibrosis. Now a team ...

Vertex Pharmaceuticals Incorporated, a biotechnology company, engages in developing and commercializing therapies for treating cystic fibrosis (CF ... includes VX-522, a CFTR mRNA therapeutic ...

In the event that two mutations are not detected by either targeted mutation analysis or full gene sequencing in an individual with high clinical suspicion for FMF, another helpful diagnostic ...

Jan. 30, 2025 — A research group has developed new advanced light-controlled tools that enable precise control of proteins in real time in living cells. This groundbreaking research opens doors ...

The locus-specific mutation frequencies for copy number variation and other structural changes are two to four orders of magnitude greater than for point mutations. HR mechanisms generally achieve ...