Duchenne Muscular Dystrophy

FDA rebuffs Entrada's attempt to get yearlong hold on DMD drug lifted
FDA rebuffs Entrada's attempt to get yearlong hold on DMD drug lifted
Fierce Biotech
Entrada Therapeutics
FDA
Duchenne Muscular Dystrophy
ENTR-601-44
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Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Sarepta enjoys early sales ramp for DMD gene therapy, maintains confidence in label expansion
Fierce Pharma
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
Elevidys
gene therapy
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Sarepta’s disappointing data put the FDA in a difficult spot
Sarepta’s disappointing data put the FDA in a difficult spot
Stat
Sarepta Therapeutics
Duchenne Muscular Dystrophy
DMD
FDA
gene therapy
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Sarepta gene therapy for Duchenne misses main goal of key study
Sarepta gene therapy for Duchenne misses main goal of key study
BioPharma Dive
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
clinical trials
FDA
Elevidys
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FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
FDA Approves Novel Corticosteroid Treatment Option for DMD Patients
BioSpace
Santhera Pharmaceuticals
Catalyst Pharmaceuticals
Agamree
FDA
DMD
Duchenne Muscular Dystrophy
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Santhera closes on first approval for DMD drug vamorolone
Santhera closes on first approval for DMD drug vamorolone
Pharmaphorum
Santhera
Duchenne Muscular Dystrophy
DMD
vamorolone
EMA
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Patient Death Likely from Virus Used to Deliver Gene-Editing Therapy: NEJM Study
Patient Death Likely from Virus Used to Deliver Gene-Editing Therapy: NEJM Study
BioSpace
NEJM
clinical trials
Duchenne Muscular Dystrophy
patient deaths
Astellas
Pfizer
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FibroGen fails again in DMD, continuing rotten run of phase 3 flops
FibroGen fails again in DMD, continuing rotten run of phase 3 flops
Fierce Biotech
FibroGen
Duchenne Muscular Dystrophy
clinical trials
pamrevlumab
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Sarepta offers early look at closely watched gene therapy launch
Sarepta offers early look at closely watched gene therapy launch
BioPharma Dive
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
Elevidys
drug launches
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Sarepta prices Duchenne gene therapy at $3.2M
Sarepta prices Duchenne gene therapy at $3.2M
BioPharma Dive
Sarepta Therapeutics
drug pricing
DMD
Duchenne Muscular Dystrophy
Elevidys
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FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
FDA Approves Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy
BioSpace
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
FDA
Elevidys
gene therapy
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FibroGen’s Duchenne drug misses mark in first large trial
FibroGen’s Duchenne drug misses mark in first large trial
BioPharma Dive
FibroGen
clinical trials
Duchenne Muscular Dystrophy
pamrevlumab
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FDA delays decision on Duchenne gene therapy and considers narrower approval
FDA delays decision on Duchenne gene therapy and considers narrower approval
BioPharma Dive
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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Sarepta Enters Uncharted Territory with Adcomm Approval
Sarepta Enters Uncharted Territory with Adcomm Approval
BioSpace
Sarepta Therapeutics
SRP-9001
DMD
Duchenne Muscular Dystrophy
FDA
gene therapy
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FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
FDA advisers narrowly vote in favor of Sarepta's DMD gene therapy
Fierce Biotech
Sarepta Therapeutics
FDA
DMD
gene therapy
Duchenne Muscular Dystrophy
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FDA reviewers flag concerns over Sarepta's gene therapy for DMD
FDA reviewers flag concerns over Sarepta's gene therapy for DMD
Yahoo/Reuters
Sarepta Therapeutics
FDA
DMD
Duchenne Muscular Dystrophy
gene therapy
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Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
Sarepta, days out from critical FDA meeting, lists questions expected at gene therapy AdComm
Fierce Biotech
Sarepta Therapeutics
FDA
SRP-9001
DMD
Duchenne Muscular Dystrophy
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Go or no go? Sarepta’s gene therapy faces scrutiny
Go or no go? Sarepta’s gene therapy faces scrutiny
EP Vantage
Sarepta Therapeutics
gene therapy
SRP-9001
Roche
DMD
Duchenne Muscular Dystrophy
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Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Sarepta shares drop as report says FDA almost rejected under-review gene therapy
Yahoo/Reuters
Sarepta Therapeutics
FDA
gene therapy
DMD
Duchenne Muscular Dystrophy
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Chasing Pfizer and Roche, Regenxbio benefits from FDA boost for DMD gene therapy
Chasing Pfizer and Roche, Regenxbio benefits from FDA boost for DMD gene therapy
Fierce Biotech
Regenxbio
adeno-associated viruses
Duchenne Muscular Dystrophy
FDA
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