Duchenne Muscular Dystrophy

Wave takes a different tack from Sarepta in Duchenne
Wave takes a different tack from Sarepta in Duchenne
EP Vantage
Wave Life Sciences
Duchenne Muscular Dystrophy
suvodirsen
Sarepta Therapeutics
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The FDA and Sarepta: a window into the real world of drug regulation
The FDA and Sarepta: a window into the real world of drug regulation
Stat
FDA
regulatory
Sarepta Therapeutics
Duchenne Muscular Dystrophy
Exondys 51
Vyondys 53
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Santhera's Duchenne effort gets a boost on positive PhIIa for alternative steroid vamorolone — though there's a lot left to prove
Santhera's Duchenne effort gets a boost on positive PhIIa for alternative steroid vamorolone — though there's a lot left to prove
Endpoints
Santhera
Duchenne Muscular Dystrophy
clinical trials
vamorolone
RaveraGen
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Sarepta now needs flawless progress for gene therapy
Sarepta now needs flawless progress for gene therapy
Biopharma Dive
Sarepta Therapeutics
FDA
Duchenne Muscular Dystrophy
Vyondys 53
gene therapy
SRP-9001
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Sarepta Duchenne drug rejected by FDA in surprise decision
Sarepta Duchenne drug rejected by FDA in surprise decision
Biopharma Dive
Sarepta Therapeutics
Duchenne Muscular Dystrophy
FDA
complete response letter
Vyondys 53
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Solid Biosciences Surges on DMD Gene Therapy Study Amendments
Solid Biosciences Surges on DMD Gene Therapy Study Amendments
Yahoo/Zacks.com
Solid Biosciences
SGT-001
Duchenne Muscular Dystrophy
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Sarepta's Lone Analyst Skeptic Takes On 22 Bulls With Renewed Warnings
Sarepta's Lone Analyst Skeptic Takes On 22 Bulls With Renewed Warnings
Bloomberg
Sarepta Therapeutics
analysts
Duchenne Muscular Dystrophy
Vyondys 53
Exondys 51
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Patient in Sarepta gene therapy trial develops serious illness; shares plunge
Patient in Sarepta gene therapy trial develops serious illness; shares plunge
Yahoo/Reuters
Sarepta Therapeutics
Duchenne Muscular Dystrophy
rhabdomyolysis
gene therapy
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Studies Show Sarepta's Exondys 51 Slows Respiratory Decline in Muscular Dystrophy Patients
Studies Show Sarepta's Exondys 51 Slows Respiratory Decline in Muscular Dystrophy Patients
BioSpace
Sarepta Therapeutics
Exondys 51
DMD
Duchenne Muscular Dystrophy
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Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study
Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study
Yahoo/Zacks.com
Pfizer
DMD
Duchenne Muscular Dystrophy
clinical trials
gene therapy
PF-06939926
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Gene Therapy vs. Gene Editing: Which Is Better for Tackling Muscular Dystrophy?
Gene Therapy vs. Gene Editing: Which Is Better for Tackling Muscular Dystrophy?
Motley Fool
gene editing
gene therapy
DMD
Duchenne Muscular Dystrophy
Vertex Pharmaceuticals
CRISPR Therapeutics
Exonics
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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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