Duchenne Muscular Dystrophy

clinical trials

As Sarepta Preps Data, FDA Lifts Hold on Solid’s Duchenne Gene Therapy

Xconomy

Mon, 06/18/18 - 11:25 am

Solid Biosciences

Sarepta’s eteplirsen spurned as European regulators turn thumbs down on controversial Duchenne MD drug

SGT-001

Endpoints

Thu, 05/3/18 - 11:14 pm

Catabasis Pharmaceuticals' Edasalonexent Slowed Duchenne Muscular Dystrophy Disease Progression Through One Year of Treatment

CP Wire

Thu, 04/26/18 - 09:49 am

Catabasis Pharmaceuticals' Edasalonexent Slowed Duchenne Muscular Dystrophy Disease Progression Through One Year of Treatment

Wed, 04/25/18 - 07:40 pm

Daiichi Sankyo concedes an early setback on Duchenne MD drug, but doubles down on development

Endpoints

Wed, 04/25/18 - 09:50 am

Daiichi Sankyo

Solid Biosciences Provides Update on Clinical Hold of Ph I/II Trial for Duchenne Muscular Dystrophy (DMD)

DS-5141

CP Wire

Wed, 04/18/18 - 09:54 am

Solid Biosciences

SGT-001

Catabasis Pharmaceuticals Reduces Workforce by 42% to Focus on Developing Edasalonexent for Duchenne Muscular Dystrophy

clinical trials

CP Wire

Wed, 04/18/18 - 09:43 am

Solid Biosciences Provides Update on Clinical Hold of Ph I/II Trial for Duchenne Muscular Dystrophy (DMD)

restructuring

layoffs

Wed, 04/18/18 - 09:40 am

Solid Biosciences

SGT-001

Catabasis Pharmaceuticals Reduces Workforce by 42% to Focus on Developing Edasalonexent for Duchenne Muscular Dystrophy

Tue, 04/17/18 - 06:50 pm

Pfizer Commences Ph Ib Trial With Mini-Dystrophin Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

CP Wire

Thu, 04/12/18 - 11:51 am

Pfizer

PF-06939926

Should Sarepta’s patents be seized by the government? Patient advocates pitch controversial drug pricing proposal

gene therapy

Endpoints

Fri, 04/6/18 - 07:39 pm

Solid Bio's DMD gene therapy graduates from partial FDA hold to full-on clinical hold

Fierce Biotech

Wed, 03/14/18 - 09:29 pm

Sarepta to seek FDA approval for its second Duchenne’s drug by year’s end

Stat

Mon, 03/12/18 - 09:37 am

Did the FDA just leave the door cracked open for PTC’s Duchenne drug after repeated slapdowns?

FDA

golodirsen

Exondys 51

Endpoints

Tue, 02/20/18 - 09:17 am

FDA

PTC Therapeutics

DMD

Sarepta halts its golodirsen Duchenne MD study in UK after patient experiences adverse event

ataluren

Endpoints

Fri, 02/9/18 - 09:35 am

golodirsen

Merck R&D chief Roger Perlmutter offers some heavyweight R&D glam to a little upstart biotech out to cure Duchenne MD

Endpoints

Tue, 01/30/18 - 09:26 am

Merck

R&D

Roger Perlmutter

Exonics Therapeutics

Anthem reverses course, agrees to cover Sarepta's Duchenne drug

BioPharma Dive

Mon, 11/13/17 - 08:46 pm

Anthem

Exondys 51

With $40M for Exonics, Duchenne’s Gene Therapy, CRISPR Race Heats Up

Xconomy

Wed, 11/8/17 - 09:24 am

Exonics Therapeutics

CRISPR

gene therapy

No Charm: PTC’s Duchenne Drug Rejected by FDA for Third Time

Xconomy

Thu, 10/26/17 - 09:25 am

PTC Therapeutics