Browsing Tag
Duchenne muscular dystrophy
26 posts
Capricor Therapeutics (NASDAQ: CAPR) reports sustained efficacy and safety in 4-year DMD trial of Deramiocel
Capricor Therapeutics reports 4-year HOPE-2 data for Deramiocel in Duchenne, showing cardiac stability and slowed muscle decline. Read how it may shape future FDA decisions.
Sarepta’s gene therapy marches on: UK clears continued dosing in Duchenne Phase 3 trial
Find out how Sarepta Therapeutics is advancing Duchenne gene therapy with UK backing for its ENVISION trial of ELEVIDYS in non-ambulatory patients.
Breakthrough data from Vatiquinone trials propels PTC Therapeutics stock to 52-week high
PTC Therapeutics Inc. (NASDAQ: PTCT) is experiencing significant momentum within the biotechnology sector following a surge in its…
Capricor Therapeutics announces promising long-term results for deramiocel deramiocel in Duchenne muscular dystrophy
Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company at the forefront of developing cell and exosome-based therapies, has shared…
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.…
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…
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…
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…
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…
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…