Duchenne Muscular Dystrophy

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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One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data
One year on, Capricor's stem cell therapy appears to help DMD patients in small study, but investors balk at the data
Endpoints
Capricor
stem cells
Duchenne Muscular Dystrophy
CAP-1002
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Wave Life Sciences hits tumultuous waters as ax comes crashing down on staffers
Wave Life Sciences hits tumultuous waters as ax comes crashing down on staffers
Fierce Biotech
Wave Life Sciences
layoffs
Duchenne Muscular Dystrophy
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FDA Flags Risk of Infection, Kidney Damage for Sarepta Duchenne Drugs
FDA Flags Risk of Infection, Kidney Damage for Sarepta Duchenne Drugs
Xconomy
Sarepta Therapeutics
Vyondys 53
Duchenne Muscular Dystrophy
FDA
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Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track
Solid Bio Restructures to Get Halted Gene Therapy Study Back on Track
Xconomy
Solid Biosciences
gene therapy
reorganization
Duchenne Muscular Dystrophy
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Roche Puts Up Historic $1.15B for Sarepta Duchenne Gene Therapy Deal
Roche Puts Up Historic $1.15B for Sarepta Duchenne Gene Therapy Deal
Xconomy
Roche
Sarepta Therapeutics
FDA
Duchenne Muscular Dystrophy
SRP-9001
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