Duchenne Muscular Dystrophy

Wave faces key test in bid to challenge Sarepta in DMD

Biopharma Dive

Sat, 11/30/19 - 11:18 pm

Wave Life Sciences

Solid Biosciences’ Duchenne gene therapy trial halted after patient suffers serious toxicity

Stat

Tue, 11/12/19 - 09:52 am

Solid Biosciences

Roche joins others in dropping myostatin inhibitor for DMD

Pharmaforum

Fri, 11/8/19 - 10:50 am

Roche

RG6206

myostatin inhibitors

Sarepta Therapeutics CEO playing nice with FDA over rejection of Duchenne drug

Stat

Thu, 11/7/19 - 10:58 pm

Sarepta competitor files with FDA for Duchenne drug

Seeking Alpha

Wed, 10/2/19 - 12:12 pm

Wave takes a different tack from Sarepta in Duchenne

EP Vantage

Thu, 09/12/19 - 10:33 am

Wave Life Sciences

suvodirsen

The FDA and Sarepta: a window into the real world of drug regulation

Stat

Tue, 09/3/19 - 04:42 pm

regulatory

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

clinical trials

vamorolone

RaveraGen

Sarepta now needs flawless progress for gene therapy

Biopharma Dive

Tue, 08/20/19 - 10:58 pm

SRP-9001

Sarepta Duchenne drug rejected by FDA in surprise decision

Biopharma Dive

Mon, 08/19/19 - 11:48 pm

complete response letter

Solid Biosciences Surges on DMD Gene Therapy Study Amendments

Yahoo/Zacks.com

Mon, 08/19/19 - 10:54 am

Solid Biosciences

SGT-001

Sarepta's Lone Analyst Skeptic Takes On 22 Bulls With Renewed Warnings

Bloomberg

Mon, 08/19/19 - 10:28 am

analysts

Patient in Sarepta gene therapy trial develops serious illness; shares plunge

Yahoo/Reuters

Thu, 08/8/19 - 11:34 pm

rhabdomyolysis

Studies Show Sarepta's Exondys 51 Slows Respiratory Decline in Muscular Dystrophy Patients

BioSpace

Wed, 07/10/19 - 04:31 pm

DMD

Pfizer's DMD Gene Therapy Shows Severe Side Effects in Study

Yahoo/Zacks.com

Mon, 07/1/19 - 10:16 am

Pfizer

DMD

clinical trials

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

DMD

UPDATED: Sarepta's Exondys 51 is not cost-effective, nor particularly beneficial for DMD patients — ICER

Vertex Pharmaceuticals

CRISPR Therapeutics

Exonics

Endpoints

Thu, 05/23/19 - 10:08 am

ICER

Solid Biosciences’ Duchenne Muscular Dystrophy Drug Has Side Effect Issues

BioSpace

Tue, 05/14/19 - 11:37 pm

Solid Biosciences

Wave down on Phase I safety data for DMD therapy

BioCentury

Tue, 04/16/19 - 11:29 pm

Wave Life Sciences