Boston Pharmaceuticals Achieves Phase 2 Success with Efimosfermin Alfa for MASH Treatment, Paving Way for Pivotal Trials

Efimosfermin alfa (BOS-580), Metabolic dysfunction-associated steatohepatitis (MASH), Non-alcoholic steatohepatitis (NASH), Phase 2 clinical trials, FGF21 analogue, Liver disease treatment, Pivotal trials

FDA Approves PTC Therapeutics’ Kebilidi, the First Gene Therapy Delivered Directly to the Brain for AADC Deficiency

PTC Therapeutics, Kebilidi, Gene Therapy, AADC Deficiency, FDA Approval, Brain-Delivered Therapy, Rare Genetic Disorder

Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy

Sarepta Therapeutics, Vesleteplirsen (SRP-5051), Duchenne Muscular Dystrophy (DMD), Exon-Skipping Therapy, FDA Feedback, Therapeutic Landscape