Tag: Duchenne muscular dystrophy
FDA approves Italfarmaco’s Duvyzat for duchenne muscular dystrophy treatment
In a significant advancement for the treatment of Duchenne muscular dystrophy (DMD), Italfarmaco S.p.A. has announced the U.S. Food and Drug Administration (FDA) approval of ... Read More
FDA advances Sarepta Therapeutics’ ELEVIDYS for Duchenne muscular dystrophy treatment
In a promising development for the treatment of rare diseases, Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a frontrunner in precision genetic medicine, announced that the U.S. Food ... Read More
IPS HEART gets third FDA pediatric rare disease designation for ISX9-CPC
IPS HEART, a private cell therapy company developing treatments for Duchenne muscular dystrophy and heart failure, announced that it has received a third Rare Pediatric ... Read More
FDA committee backs approval of Sarepta Therapeutics’ SRP-9001 for DMD
Sarepta Therapeutics’ SRP-9001 (delandistrogene moxeparvovec) has received the backing of the Cellular, Tissue and Gene Therapies Advisory Committee (CTGTAC) of the US Food and Drug ... Read More
Sarepta Therapeutics to seek FDA accelerated approval for SRP-9001 in DMD
Sarepta Therapeutics said that it plans to file a biologics license application (BLA) with the US Food and Drug Administration (FDA) to seek accelerated approval ... Read More
ReveraGen, Santhera get FDA grant for BMD trial of vamorolone
ReveraGen Biopharma and Santhera Pharmaceuticals have secured a $1.2 million grant for funding their clinical trial of vamorolone in Becker muscular dystrophy (BMD) from the ... Read More
PTC Therapeutics wins Russian approval for Translarna for nmDMD
US-based PTC Therapeutics has secured marketing approval in Russia for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The approval for ... Read More
Roche signs $2.85bn deal with Sarepta for SRP-9001 DND gene therapy
Roche has signed a licensing deal worth up to $2.85 billion with Sarepta Therapeutics for the exclusive ex-US commercial rights to the latter’s SRP-9001 (AAVrh74.MHCK7.micro-dystrophin), ... Read More
Astellas Pharma to acquire US gene therapy company Audentes Therapeutics
Astellas acquisition of Audentes: Japanese pharma company Astellas Pharma has agreed to acquire US gene therapy company Audentes Therapeutics in an all-cash deal worth about ... Read More
Catabasis’ PolarisDMD clinical trial for edasalonexent completes enrollment
US biopharma company Catabasis Pharmaceuticals has wrapped up enrollment for the phase 3 PolarisDMD clinical trial designed to evaluate edasalonexent, an NF-kB inhibitor, in Duchenne ... Read More