Now, research reported in Nature has identified a small molecule known as PTC124 as a potential novel treatment to target the specific genetic defects that underlie a wide range of these diseases.

Dr. Steven Rowe is the international PI of the VX-809 study (targeting DF508 CFTR, the most common cause of CF) Dr. Steven Rowe is the Co-PI of the international study of PTC124 (targeting premature ...

Objective: To review and evaluate cystic fibrosis transmembrane conductance regulator (CFTR) modulators for the treatment of cystic fibrosis (CF). Data Sources: Literature was accessed through ...

A lawsuit filed by Cheri Gunvalson to force privately held biotech PTC Therapeutics to provide her son with access to its experimental drug could have widespread ramifications for the industry.

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1 Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, UK Objective With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand ...