Duchenne Muscular Dystrophy

Sarepta's DMD gene therapy fails phase 2 motor function test, sinking stock
Sarepta's DMD gene therapy fails phase 2 motor function test, sinking stock
Fierce Biotech
Sarepta Therapeutics
Duchenne Muscular Dystrophy
clinical trials
SRP-9001
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Pfizer beats Sarepta to the start of first late-stage Duchenne gene therapy trial
Pfizer beats Sarepta to the start of first late-stage Duchenne gene therapy trial
BioPharma Dive
Pfizer
Sarepta Therapeutics
clinical trials
Duchenne Muscular Dystrophy
gene therapy
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Sarepta shares first results on next-generation Duchenne drug
Sarepta shares first results on next-generation Duchenne drug
BioPharma Dive
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-5051
Exondys 51
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Could Eli Lilly and Precision BioSciences Make Breakthroughs for DMD?
Could Eli Lilly and Precision BioSciences Make Breakthroughs for DMD?
BioSpace
Eli Lilly
Precision Biosciences
Duchenne Muscular Dystrophy
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Santhera axes half its employees and sees CMO depart after PhIII DMD fail
Santhera axes half its employees and sees CMO depart after PhIII DMD fail
Endpoints
Santhera
reorganization
Duchenne Muscular Dystrophy
vamorolone
layoffs
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Santhera flunks DMD phase 3, prompting restructuring and withdrawal of approval application
Santhera flunks DMD phase 3, prompting restructuring and withdrawal of approval application
Fierce Biotech
Santhera Pharmaceuticals
clinical trials
Duchenne Muscular Dystrophy
idebenone
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FDA Grants Fast Track Designation to Pfizer's DMD Gene Therapy
FDA Grants Fast Track Designation to Pfizer's DMD Gene Therapy
BioSpace
Pfizer
fast track
FDA
Duchenne Muscular Dystrophy
PF-06939926
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FDA Lifts Clinical Hold on Solid Biosciences’ DMD Gene Therapy Trial
FDA Lifts Clinical Hold on Solid Biosciences’ DMD Gene Therapy Trial
BioSpace
Solid Biosciences
FDA
clinical trials
gene therapy
Duchenne Muscular Dystrophy
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New data bolsters Sarepta gene therapies for two deadly muscle diseases
New data bolsters Sarepta gene therapies for two deadly muscle diseases
Biopharma Dive
Sarepta Therapeutics
gene therapy
Duchenne Muscular Dystrophy
Limb Girdle Muscular Dystrophy
SRP-9001
SRP-9003
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Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent
Sarepta faces another gene therapy hiccup as Regenxbio sues over Jim Wilson's patent
Endpoints
Sarepta Therapeutics
gene therapy
Regenxbio
Duchenne Muscular Dystrophy
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Sarepta cites 'overburdened' FDA as factor in DMD gene therapy delay
Sarepta cites 'overburdened' FDA as factor in DMD gene therapy delay
Fierce Biotech
Sarepta Therapeutics
Duchenne Muscular Dystrophy
gene therapy
FDA
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Sarepta shares fall on potential regulatory delay for experimental Duchenne drug
Sarepta shares fall on potential regulatory delay for experimental Duchenne drug
Marketwatch
Sarepta Therapeutics
Duchenne Muscular Dystrophy
SRP-9001
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Santhera bags DMD drug in 3-way deal with Idorsia and ReveraGen
Santhera bags DMD drug in 3-way deal with Idorsia and ReveraGen
Fierce Biotech
Santhera
vamorolone
Duchenne Muscular Dystrophy
Idorsia
ReveraGen
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Sarepta Therapeutics Gets Fast-Track FDA Review For Muscular Dystrophy Drug
Sarepta Therapeutics Gets Fast-Track FDA Review For Muscular Dystrophy Drug
Investors Business Daily
Sarepta Therapeutics
FDA
fast track
Duchenne Muscular Dystrophy
casimersen
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FDA gives speedy approval to another Duchenne drug
FDA gives speedy approval to another Duchenne drug
BioPharma Dive
FDA
NS Pharma
Japan
Duchenne Muscular Dystrophy
Viltepso
viltolarsen
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FDA keeps Solid Bio's troubled Duchenne MD gene therapy on clinical lockdown in latest setback
FDA keeps Solid Bio's troubled Duchenne MD gene therapy on clinical lockdown in latest setback
Endpoints
Solid Biosciences
Duchenne Muscular Dystrophy
FDA
gene therapy
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5 FDA approval decisions to watch in the 3rd quarter
5 FDA approval decisions to watch in the 3rd quarter
BioPharma Dive
FDA
Gilead Sciences
filgotinib
rheumatoid arthritis
GSK
belantamab mafodotin
Multiple Myeloma
NS Pharma
viltolarsen
Duchenne Muscular Dystrophy
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Sarepta bags drug to fully unlock DMD gene therapy opportunity
Sarepta bags drug to fully unlock DMD gene therapy opportunity
Fierce Biotech
Sarepta Therapeutics
Duchenne Muscular Dystrophy
Hansa Biopharma
gene therapy
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OptumRx: The 3 drugs that will have the biggest impact on payers, patients in 2020
OptumRx: The 3 drugs that will have the biggest impact on payers, patients in 2020
Beckers Hospital Review
OptumRx
Roche
PTC Therapeutics
risdiplam
SMA
NS Pharma
viltolarsen
Duchenne Muscular Dystrophy
Immunomedics
Trodelvy
triple negative breast cancer
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Safety questions linger as Pfizer preps Duchenne gene therapy for final test
Safety questions linger as Pfizer preps Duchenne gene therapy for final test
BioPharma Dive
Pfizer
gene therapy
Duchenne Muscular Dystrophy
PF-06939926
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