Pharma Focus Asia

U.S. Panel Finds Sarepta Muscular Dystrophy Drug not Proven Effective

Tuesday, April 26, 2016

An experimental drug to treat Duchenne muscular dystrophy, a devastating degenerative disease that mostly affects young boys, was not supported by enough evidence to prove it is effective, a U.S. advisory panel concluded on Monday, decreasing the drug's chances of approval

The vote followed an emotional meeting at a hotel in Hyattsville, Maryland, in which hundreds of patients and their advocates gathered to urge the Food and Drug Administration to approve the drug, Sarepta Therapeutics Inc's eteplirsen, saying their children had benefited from it.

 

Source : reuters.com

magazine-slider-imageMFA + MMA 2024CPHI Chine || PMEC China 2024Asia Healthcare Week 2024Advance DoE WorkshopNitrosamine Advance Workshop 2024CPHI Korea 2024CHEMICAL INDONESIA 2024INALAB 2024 Thermo Scientific - DynaDrive and DynaSpinDigital Health Asia 2024Rehab Expo 2024ISPE Singapore Affiliate Conference & Exhibition 20242024 PDA Pharmaceutical Manufacturing & Quality Conference2024 PDA Cell and Gene Pharmaceutical Products Conference 2024 PDA Aseptic Manufacturing Excellence Conference2024 PDA Aseptic Processing of Biopharmaceuticals ConferencePharma Quality Excellence Awards 20244th Annual Pharma GMP Quality Management 20243rd World ADC Asia 2024LogiPharma Asia 2024