Duchenne Muscular Dystrophy

The FDA and Sarepta: a window into the real world of drug regulation
Stat
Tue, 09/3/19 - 04:42 pm
FDA
regulatory
Sarepta Therapeutics
Duchenne Muscular Dystrophy
Exondys 51
Vyondys 53
Santhera's Duchenne effort gets a boost on positive PhIIa for alternative steroid vamorolone — though there's a lot left to prove
Endpoints
Tue, 08/27/19 - 11:01 pm
Santhera
Duchenne Muscular Dystrophy
clinical trials
vamorolone
RaveraGen
Sarepta now needs flawless progress for gene therapy
Biopharma Dive
Tue, 08/20/19 - 10:58 pm
Sarepta Therapeutics
FDA
Duchenne Muscular Dystrophy
Vyondys 53
gene therapy
SRP-9001
Sarepta Duchenne drug rejected by FDA in surprise decision
Biopharma Dive
Mon, 08/19/19 - 11:48 pm
Sarepta Therapeutics
Duchenne Muscular Dystrophy
FDA
complete response letter
Vyondys 53
Solid Biosciences Surges on DMD Gene Therapy Study Amendments
Yahoo/Zacks.com
Mon, 08/19/19 - 10:54 am
Solid Biosciences
SGT-001
Duchenne Muscular Dystrophy
Sarepta's Lone Analyst Skeptic Takes On 22 Bulls With Renewed Warnings
Bloomberg
Mon, 08/19/19 - 10:28 am
Sarepta Therapeutics
analysts
Duchenne Muscular Dystrophy
Vyondys 53
Exondys 51
Patient in Sarepta gene therapy trial develops serious illness; shares plunge
Yahoo/Reuters
Thu, 08/8/19 - 11:34 pm
Sarepta Therapeutics
Duchenne Muscular Dystrophy
rhabdomyolysis
gene therapy
Studies Show Sarepta's Exondys 51 Slows Respiratory Decline in Muscular Dystrophy Patients
BioSpace
Wed, 07/10/19 - 04:31 pm
Sarepta Therapeutics
Exondys 51
DMD
Duchenne Muscular Dystrophy
Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study
Yahoo/Zacks.com
Mon, 07/1/19 - 10:16 am
Pfizer
DMD
Duchenne Muscular Dystrophy
clinical trials
gene therapy
PF-06939926
Gene Therapy vs. Gene Editing: Which Is Better for Tackling Muscular Dystrophy?
Motley Fool
Sun, 06/9/19 - 01:41 pm
gene editing
gene therapy
DMD
Duchenne Muscular Dystrophy
Vertex Pharmaceuticals
CRISPR Therapeutics
Exonics
UPDATED: Sarepta's Exondys 51 is not cost-effective, nor particularly beneficial for DMD patients — ICER
Endpoints
Thu, 05/23/19 - 10:08 am
Duchenne Muscular Dystrophy
ICER
Sarepta Therapeutics
Exondys 51
Solid Biosciences’ Duchenne Muscular Dystrophy Drug Has Side Effect Issues
BioSpace
Tue, 05/14/19 - 11:37 pm
Solid Biosciences
Duchenne Muscular Dystrophy
Wave down on Phase I safety data for DMD therapy
BioCentury
Tue, 04/16/19 - 11:29 pm
Wave Life Sciences
Duchenne Muscular Dystrophy
clinical trials
suvodirsen
Sarepta to seek speedy approval of third DMD drug
Biopharma Dive
Thu, 03/28/19 - 07:57 pm
Sarepta Therapeutics
FDA
Duchenne Muscular Dystrophy
casimersen
Upcoming events – Fibrogen and Alnylam look to broaden their horizons
EP Vantage
Sat, 03/2/19 - 12:44 pm
FibroGen
roxadustat
pamrevlumab
Duchenne Muscular Dystrophy
givosiran
Alnylam
CRISPR corrects DMD in mice for a year in Sarepta-supported study
Fierce Biotech
Mon, 02/18/19 - 11:29 am
Duke University
Sarepta Therapeutics
CRISPR
gene editing
DMD
Duchenne Muscular Dystrophy
Solid Bio is hit by another setback as initial biopsies spotlight a flop for Duchenne MD
Endpoints
Thu, 02/7/19 - 09:37 am
Solid Biosciences
clinical trials
Duchenne Muscular Dystrophy
SGT-001
Suvodirsen For Duchenne Muscular Dystrophy Selected for FDA Complex Innovative Trial Designs Pilot Program
CP Wire
Thu, 01/3/19 - 09:43 am
suvodirsen
Wave Life Sciences
Duchenne Muscular Dystrophy
muscular dystrophy
Suvodirsen For Duchenne Muscular Dystrophy Selected for FDA Complex Innovative Trial Designs Pilot Program
Thu, 01/3/19 - 09:41 am
suvodirsen
Wave Life Sciences
Duchenne Muscular Dystrophy
muscular dystrophy
15 For ’19: Key Clinical Data To Watch For Next Year (Part 1)
Xconomy
Mon, 12/17/18 - 09:44 am
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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