Duchenne Muscular Dystrophy

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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
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
Flag link:
5 FDA decisions to watch in the second quarter
5 FDA decisions to watch in the second quarter
BioPharma Dive
FDA
Sarepta Therapeutics
Duchenne Muscular Dystrophy
Biogen
tofersen
ALS
Seres Therapeutics
C. difficile
Pfizer
GSK
vaccines
RSV
Regeneron
Eylea
Flag link:
Vertex flexes its dealmaking muscle
Vertex flexes its dealmaking muscle
EP Vantage
Vertex Pharmaceuticals
Entrada Therapeutics
Tevard Biosciences
Duchenne Muscular Dystrophy
Flag link:
FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy
FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy
BioSpace
FDA
Sarepta Therapeutics
DMD
gene therapy
Duchenne Muscular Dystrophy
SRP-9001
Flag link:
Duchenne muscular dystrophy: major trials and events to watch in 2023
Duchenne muscular dystrophy: major trials and events to watch in 2023
Clinical Trials Arena
DMD
Duchenne Muscular Dystrophy
clinical trials
gene therapy
FDA
Sarepta Therapeutics
SRP-9001
FibroGen
pamrevlumab
Capricor
CAP-1002
Santhera
ReveraGen
vamorolone
Flag link:
Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy
Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy
Biopharma Reporter
Sarepta Therapeutics
DMD
Duchenne Muscular Dystrophy
gene therapy
FDA
Flag link:
Sarepta's DMD gene therapy inches closer to market as FDA declines to hold advisory meeting
Sarepta's DMD gene therapy inches closer to market as FDA declines to hold advisory meeting
Fierce Biotech
FDA
Sarepta Therapeutics
SRP-9001
gene therapy
DMD
Duchenne Muscular Dystrophy
Flag link:

Pages