Duchenne Muscular Dystrophy

UPDATED: Sarepta's Exondys 51 is not cost-effective, nor particularly beneficial for DMD patients — ICER
UPDATED: Sarepta's Exondys 51 is not cost-effective, nor particularly beneficial for DMD patients — ICER
Endpoints
Duchenne Muscular Dystrophy
ICER
Sarepta Therapeutics
Exondys 51
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Solid Biosciences’ Duchenne Muscular Dystrophy Drug Has Side Effect Issues
Solid Biosciences’ Duchenne Muscular Dystrophy Drug Has Side Effect Issues
BioSpace
Solid Biosciences
Duchenne Muscular Dystrophy
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Wave down on Phase I safety data for DMD therapy
Wave down on Phase I safety data for DMD therapy
BioCentury
Wave Life Sciences
Duchenne Muscular Dystrophy
clinical trials
suvodirsen
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Sarepta to seek speedy approval of third DMD drug
Sarepta to seek speedy approval of third DMD drug
Biopharma Dive
Sarepta Therapeutics
FDA
Duchenne Muscular Dystrophy
casimersen
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Upcoming events – Fibrogen and Alnylam look to broaden their horizons
Upcoming events – Fibrogen and Alnylam look to broaden their horizons
EP Vantage
FibroGen
roxadustat
pamrevlumab
Duchenne Muscular Dystrophy
givosiran
Alnylam
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CRISPR corrects DMD in mice for a year in Sarepta-supported study
CRISPR corrects DMD in mice for a year in Sarepta-supported study
Fierce Biotech
Duke University
Sarepta Therapeutics
CRISPR
gene editing
DMD
Duchenne Muscular Dystrophy
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Solid Bio is hit by another setback as initial biopsies spotlight a flop for Duchenne MD
Solid Bio is hit by another setback as initial biopsies spotlight a flop for Duchenne MD
Endpoints
Solid Biosciences
clinical trials
Duchenne Muscular Dystrophy
SGT-001
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Suvodirsen For Duchenne Muscular Dystrophy Selected for FDA Complex Innovative Trial Designs Pilot Program
Suvodirsen For Duchenne Muscular Dystrophy Selected for FDA Complex Innovative Trial Designs Pilot Program
CP Wire
suvodirsen
Wave Life Sciences
Duchenne Muscular Dystrophy
muscular dystrophy
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Suvodirsen For Duchenne Muscular Dystrophy Selected for FDA Complex Innovative Trial Designs Pilot Program
suvodirsen
Wave Life Sciences
Duchenne Muscular Dystrophy
muscular dystrophy
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15 For ’19: Key Clinical Data To Watch For Next Year (Part 1)
15 For ’19: Key Clinical Data To Watch For Next Year (Part 1)
Xconomy
clinical trials
Alzheimer's disease
Biogen
Duchenne Muscular Dystrophy
Pfizer
Solid Biosciences
Sarepta Therapeutics
hemophilia A
Biomarin
Multiple Myeloma
Celgene
Bluebird Bio
Amgen
solid tumors
Bristol-Myers Squibb
Nektar Therapeutics
age-related macular degeneration
Regenxbio
SAGE Therapeutics
MorphoSys
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Increase collaboration to widen access to DMD drugs, trials and care
Increase collaboration to widen access to DMD drugs, trials and care
Pharmaforum
Duchenne Muscular Dystrophy
clinical trials
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Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy
Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy
Forbes
Sarepta Therapeutics
Duchenne Muscular Dystrophy
gene therapy
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Catabasis Pharmaceuticals Initiates Ph III Trial for Promising Duchenne Muscular Dystrophy Drug
Catabasis Pharmaceuticals Initiates Ph III Trial for Promising Duchenne Muscular Dystrophy Drug
CP Wire
Catabasis
edasalonexent
Duchenne Muscular Dystrophy
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Catabasis Pharmaceuticals Initiates Ph III Trial for Promising Duchenne Muscular Dystrophy Drug
Catabasis
edasalonexent
Duchenne Muscular Dystrophy
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FDA lifts clinical hold on Sarepta's Duchenne MD gene therapy, clearing the way to a quick launch of a pivotal trial
FDA lifts clinical hold on Sarepta's Duchenne MD gene therapy, clearing the way to a quick launch of a pivotal trial
Endpoints
Sarepta Therapeutics
FDA
gene therapy
Duchenne Muscular Dystrophy
clinical trials
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Europe Advisory Panel Rejects Sarepta’s Duchenne Drug Once Again
Europe Advisory Panel Rejects Sarepta’s Duchenne Drug Once Again
Xconomy
Europe
Sarepta Therapeutics
Duchenne Muscular Dystrophy
Exondys 51
eteplirsen
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Researchers Use CRISPR to Treat Duchenne Muscular Dystrophy in Dogs
Researchers Use CRISPR to Treat Duchenne Muscular Dystrophy in Dogs
BioSpace
DMD
Duchenne Muscular Dystrophy
CRISPR
drug development
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Pfizer Ends Trials for Domagrozumab in Drug for Duchenne Muscular Dystrophy
Pfizer Ends Trials for Domagrozumab in Drug for Duchenne Muscular Dystrophy
CP Wire
Pfizer
domagrozumab
Duchenne Muscular Dystrophy
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Pfizer Ends Trials for Domagrozumab in Drug for Duchenne Muscular Dystrophy
Pfizer
domagrozumab
Duchenne Muscular Dystrophy
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Mallinckrodt Starts Phase II Duchenne Muscular Dystrophy Study but Will They Meet Recruitment Targets?
Mallinckrodt Starts Phase II Duchenne Muscular Dystrophy Study but Will They Meet Recruitment Targets?
Xtalks
Mallinckrodt
MNK-1411
Duchenne Muscular Dystrophy
clinical trials
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