Duchenne Muscular Dystrophy

Sarepta pulls plug on Duchenne exon-skipping drug
Pharmaphorum
Thu, 11/7/24 - 11:09 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
vesleteplirsen
PTC Therapeutics Faces Setback on Translarna Approval
Tip Ranks
Fri, 10/18/24 - 11:31 am
PTC Therapeutics
EMA
Europe
Translarna
Duchenne Muscular Dystrophy
Capricor cell therapy improves cardiac and upper limb function in phase 2 Duchenne muscular dystrophy victory lap
Fierce Biotech
Sun, 10/13/24 - 11:27 am
Capricor
DMD
clinical trials
cell therapy
Duchenne Muscular Dystrophy
deramiocel
Wave surfs DMD success to regulators' doors, sending stock up
Fierce Biotech
Tue, 09/24/24 - 11:45 am
Wave Life Sciences
DMD
Duchenne Muscular Dystrophy
clinical trials
FDA designations boost AI-Driven cell therapy approach for Duchenne muscular dystrophy
Biopharma Reporter
Tue, 09/17/24 - 09:53 am
Somite
FDA
pediatrics
cell replacement therapy
artificial intelligence
DMD
Duchenne Muscular Dystrophy
DMD clinical studies evolve with emphasis on building designs around relevant endpoints
Clinical Trials Arena
Fri, 08/30/24 - 11:24 am
DMD
Duchenne Muscular Dystrophy
clinical trials
Pfizer gene therapy for Duchenne fails to meet goals of key trial
BioPharma Dive
Wed, 06/12/24 - 11:32 pm
Pfizer
Duchenne Muscular Dystrophy
DMD
clinical trials
Ciffreo
FDA approves Italfarmaco’s Duvyzat for DMD treatment
Pharmaceutical Business Review
Fri, 03/22/24 - 09:38 am
Italfarmaco
FDA
Duvyzat
DMD
Duchenne Muscular Dystrophy
Regenxbio data suggest ‘niche’ in Duchenne gene therapy
BioPharma Dive
Thu, 03/7/24 - 11:05 am
Regenxbio
Duchenne Muscular Dystrophy
DMD
RGX-202
Sarepta Therapeutics
Elevidys
Sarepta’s Duchenne Gene Therapy Elevidys Nabs $200M on Strong Demand
BioSpace
Thu, 02/29/24 - 11:00 am
Sarepta Therapeutics
gene therapy
Elevidys
earnings
Duchenne Muscular Dystrophy
FDA to review Sarepta's Duchenne gene therapy for traditional approval
Reuters
Fri, 02/16/24 - 11:38 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
gene therapy
FDA
Elevidys
Santhera debuts DMD drug Agamree in Germany
Pharmaphorum
Mon, 01/15/24 - 09:37 am
Santhera
Duchenne Muscular Dystrophy
DMD
Agamree
Germany
Dyne's shares surge after promising data on muscle disorder treatment
Reuters
Wed, 01/3/24 - 09:33 am
Dyne Therapeutics
clinial trials
DYNE-101
myotonic muscular dystrophy
Duchenne Muscular Dystrophy
FDA rebuffs Entrada's attempt to get yearlong hold on DMD drug lifted
Fierce Biotech
Wed, 11/22/23 - 12:03 pm
Entrada Therapeutics
FDA
Duchenne Muscular Dystrophy
ENTR-601-44
Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Fierce Pharma
Thu, 11/2/23 - 05:15 pm
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
Elevidys
gene therapy
Sarepta’s disappointing data put the FDA in a difficult spot
Stat
Tue, 10/31/23 - 10:16 am
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
FDA
gene therapy
Sarepta gene therapy for Duchenne misses main goal of key study
BioPharma Dive
Mon, 10/30/23 - 08:17 pm
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
clinical trials
FDA
Elevidys
FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
BioSpace
Fri, 10/27/23 - 11:47 am
Santhera Pharmaceuticals
Catalyst Pharmaceuticals
Agamree
FDA
DMD
Duchenne Muscular Dystrophy
Santhera closes on first approval for DMD drug vamorolone
Pharmaphorum
Fri, 10/13/23 - 10:23 am
Santhera
Duchenne Muscular Dystrophy
DMD
vamorolone
EMA
Patient Death Likely from Virus Used to Deliver Gene-Editing Therapy: NEJM Study
BioSpace
Thu, 09/28/23 - 10:33 am
NEJM
clinical trials
Duchenne Muscular Dystrophy
patient deaths
Astellas
Pfizer

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