Duchenne Muscular Dystrophy

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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Sarepta to Expedite Timeline for Muscular Dystrophy Gene Therapy
Sarepta to Expedite Timeline for Muscular Dystrophy Gene Therapy
BioSpace
Sarepta Therapeutics
SRP-9001
Duchenne Muscular Dystrophy
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Sarepta shares buoyed by pledge to seek an early swift review for Duchenne gene therapy
Sarepta shares buoyed by pledge to seek an early swift review for Duchenne gene therapy
Fierce Biotech
Sarepta Therapeutics
SRP-9001
Duchenne Muscular Dystrophy
Roche
accelerated approvals
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Myocarditis case mars Sarepta DMD gene therapy readout
Myocarditis case mars Sarepta DMD gene therapy readout
Pharmaforum
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
SRP-9001
myocarditis
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Another Duchenne clinical hold gets lifted, this time at Dyne Therapeutics
Another Duchenne clinical hold gets lifted, this time at Dyne Therapeutics
Endpoints
Duchenne Muscular Dystrophy
clinical trials
Dyne Therapeutics
DYNE-251
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As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators
As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators
MedCity News
Duchenne Muscular Dystrophy
Italy
Ifalfarmco
givinostat
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FDA suspends US testing of Sarepta Duchenne drug over safety concerns
FDA suspends US testing of Sarepta Duchenne drug over safety concerns
BioPharma Dive
FDA
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-5051
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PTC Ready to Take Another Shot at FDA Approval of DMD Drug
PTC Ready to Take Another Shot at FDA Approval of DMD Drug
BioSpace
PTC Therapeutics
Translarna
FDA
Duchenne Muscular Dystrophy
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Pfizer, Sarepta team with fellow DMD gene therapy makers to get to the bottom of adverse events
Pfizer, Sarepta team with fellow DMD gene therapy makers to get to the bottom of adverse events
Fierce Biotech
Duchenne Muscular Dystrophy
gene therapy
Pfizer
Sarepta Therapeutics
Genethon
Solid Biosciences
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With Sarepta in its sights, PepGen prices IPO to fund DMD trials
With Sarepta in its sights, PepGen prices IPO to fund DMD trials
Fierce Biotech
PepGen
IPOs
Sarepta Therapeutics
Duchenne Muscular Dystrophy
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FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards
FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards
BioPharma Dive
Pfizer
gene therapy
Duchenne Muscular Dystrophy
clinical trial
FDA
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Roche delays DMD drug deadline, decides to rejig MS trials because of Ukraine war
Roche delays DMD drug deadline, decides to rejig MS trials because of Ukraine war
Fierce Biotech
Roche
Ocrevus
clinical trials
Duchenne Muscular Dystrophy
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