Catabasis Pharmaceuticals Inc. a clinical-stage drug development company built on a pathway pharmacology technology platform today announced that dosing of the first patient has been initiated in the MoveDMD trial a Phase 1/2 trial of CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD).
Santhera Pharmaceuticals (SIX: SANN) announces that it has discussed its plans to submit a New Drug Application (NDA) for Raxone®/Catena® (idebenone) in DMD with the US FDA in a pre-NDA meeting.
Capricor Therapeutics, Inc. (Nasdaq:CAPR), a biotechnology company focused on developing novel therapeutics for the treatment of cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug Application (IND) for the clinical development of CAP-1002 for the treatment of patients with Duchenne Muscular Dystrophy (DMD) related cardiomyopathy.
BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping.
The Duchenne community is celebrating another promising step toward an approved treatment. On May 19 Sarepta Therapeutics announced plans to submit a rolling NDA for eteplirsen with the final […]
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, today announced that the Company held a pre-New Drug Application (NDA) meeting with the U.S. Food and Drug Administration (FDA) regarding its lead product candidate, eteplirsen, for the treatment of Duchenne muscular dystrophy (DMD).
For some people it takes a trophy or medal to denote a champion. For those in the fight against Duchenne Muscular Dystrophy, there are all kinds of champions and […]
Guest blog by Jennifer Wallace, physical therapist and project manager of CureDuchenne Cares. Yesterday was an important day for me since it was my last day working in a local […]
CureDuchenne, a nonprofit that raises awareness and funds research to find a cure for Duchenne muscular dystrophy, joined forces with Jennifer Wallace, physical therapist and founder of the Duchenne Therapy […]
