Duchenne Muscular Dystrophy

Vertex flexes its dealmaking muscle
Vertex flexes its dealmaking muscle
EP Vantage
Vertex Pharmaceuticals
Entrada Therapeutics
Tevard Biosciences
Duchenne Muscular Dystrophy
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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
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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
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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
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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
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Vertex tiptoes further into Duchenne research with Tevard deal
Vertex tiptoes further into Duchenne research with Tevard deal
BioPharma Dive
Vertex Pharmaceuticals
Duchenne Muscular Dystrophy
DMD
Tevard Biosciences
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Duchenne muscular dystrophy’s progress so far in 2023
Duchenne muscular dystrophy’s progress so far in 2023
Clinical Trials Arena
Duchenne Muscular Dystrophy
DMD
Santhera Pharmaceuticals
ReveraGen
vamorolone
Regenxbio
RGX-202
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FDA accepts Santhera, ReveraGen’s vamorolone NDA to treat DMD
FDA accepts Santhera, ReveraGen’s vamorolone NDA to treat DMD
Pharmaceutical Business Review
Santhera Pharmaceuticals
ReveraGen
vamorolone
Duchenne Muscular Dystrophy
FDA
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Sarepta marches forward with its potential DMD gene therapy and an expanded Catalent manufacturing deal
Sarepta marches forward with its potential DMD gene therapy and an expanded Catalent manufacturing deal
Endpoints
Sarepta Therapeutics
SRP-9001
gene therapy
Duchenne Muscular Dystrophy
CDMOs
Catalent
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Fresh from Vertex payday, Entrada's DMD plans are derailed by FDA hold
Fresh from Vertex payday, Entrada's DMD plans are derailed by FDA hold
Fierce Biotech
Entrada Therapeutics
Vertex Pharmaceuticals
FDA
clinical trials
Duchenne Muscular Dystrophy
myotonic muscular dystrophy
ENTR-601-44
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FDA grants speedy review to Sarepta’s Duchenne gene therapy
FDA grants speedy review to Sarepta’s Duchenne gene therapy
BioPharma Dive
Sarepta Therapeutics
SRP-9001
FDA
Duchenne Muscular Dystrophy
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FDA preps for DMD drug generics as Sarepta has yet to finish its confirmatory trial
FDA preps for DMD drug generics as Sarepta has yet to finish its confirmatory trial
Endpoints
Sarepta Therapeutics
generics
Duchenne Muscular Dystrophy
FDA
eteplirsen
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Only Patient in N-of-1 CRISPR Trial for Duchenne Muscular Dystrophy Dies
Only Patient in N-of-1 CRISPR Trial for Duchenne Muscular Dystrophy Dies
BioSpace
CRISPR
clinical trials
patient deaths
Duchenne Muscular Dystrophy
CRD-TMH-001
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Sarepta preps $1.2B war chest as DMD gene therapy nears accelerated FDA nod
Sarepta preps $1.2B war chest as DMD gene therapy nears accelerated FDA nod
Fierce Biotech
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-9001
gene therapy
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Santhera seeks speedy FDA review of Duchenne drug vamorolone
Santhera seeks speedy FDA review of Duchenne drug vamorolone
Pharmaphorum
Santhera
Duchenne Muscular Dystrophy
vamorolone
FDA
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Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy
Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy
BioPharma Dive
Sarepta Therapeutics
Duchenne Muscular Dystrophy
FDA
gene therapy
SRP-9001
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Pepgen aims to overtake Sarepta
Pepgen aims to overtake Sarepta
EP Vantage
Sarepta Therapeutics
PepGen
Duchenne Muscular Dystrophy
PGN-ED051
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Eyeing accelerated approval for Duchenne gene therapy, Sarepta hopes to bring in $1B in bid for profitability
Eyeing accelerated approval for Duchenne gene therapy, Sarepta hopes to bring in $1B in bid for profitability
Endpoints
Sarepta Therapeutics
Duchenne Muscular Dystrophy
gene therapy
FDA
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After serious side effect, Sarepta and FDA realign on next-gen Duchenne drug with trial hold lifted
After serious side effect, Sarepta and FDA realign on next-gen Duchenne drug with trial hold lifted
Endpoints
Sarepta Therapeutics
Duchenne Muscular Dystrophy
vesleteplirsen
clinical trials
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Did Sarepta Therapeutics Just Unveil a Breakthrough?
Did Sarepta Therapeutics Just Unveil a Breakthrough?
TheStreet.com
Sarepta Therapeutics
Duchenne Muscular Dystrophy
FDA
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