Entrada Therapeutics has dosed its first participant in their Phase 1 clinical trial of ENTR-601-44 for Duchenne muscular dystrophy.
Continue readingEntrada Therapeutics: First Participant Dosed in Phase 1 Clinical Trial of ENTR-601-44
World Duchenne Organization: EMA’s Committee for Medicinal Products for Human Use (CHMP) is not renewing the Marketing Authorisation for Translarna (ataluren)
The EMA’s human medicines committee (CHMP) has recommended not to renew the Marketing Authorization for Translarna (ataluren), a medicine for treating patients with Duchenne muscular dystrophy (DMD).
Continue readingItalfarmaco: Receives EMA Validation of Marketing Authorization Application for Givinostat
Italfamaraco has submitted the Marketing Authorization Application (MAA) for Givinostat as a potential treatment for Duchenne muscular dystrophy to the European Medicine Agency (EMA) and that the EMA has started its regulatory review process.
Continue readingFibroGen: Topline Results from LELANTOS-2 Phase 3 Study
FibroGen has announced topline results of the Phase 3 LELANTOS-2 trial of pamrevlumab, their investigational anti-fibrotic monoclonal antibody, for ambulatory patients with Duchenne muscular dystrophy.
Continue readingAvidity Biosciences: Receives FDA Orphan Drug Designation for AOC 1044
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy.
Continue readingEdgewise Therapeutics: DMD/BMD Clinical Trial Updates
Edgewise Therapeutics has provided an updated on Duchenne and Becker muscular dystrophy clinical trials.
Continue readingNS Pharma: FDA Grants Orphan Drug Designation to NS-089/NCNP-02
The FDA has granted Orphan Drug Designation to NS-089/NCNP-02 for the treatment of Duchenne muscular dystrophy.
Continue readingSatellos Bioscience: Receives Orphan Drug Designation and Rare Pediatric Disease Designation for SAT-3153 in the United States
Satellos Bioscience has received Orphan Drug Designation and Rare Pediatric Disease Designation for SAT-3153 in the United States.
Continue readingCatalyst Pharmaceuticals: Exclusive North American License For Vamorolone
Catalyst Pharmaceuticals has acquired the exclusive North American license for vamorolone for Duchenne muscular dystrophy from Santhera Pharmaceuticals.
Continue readingCasimir: ARISE Study Canadian Recruitment
Casimir is currently recruiting Canadians with Duchenne for their ARISE remote study evaluating the performance of the Duchenne Video Assessment (DVA) tool to assess quality of movement in Duchenne.
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