Duchenne Muscular Dystrophy

Sarepta to Expedite Timeline for Muscular Dystrophy Gene Therapy

BioSpace

Thu, 08/4/22 - 10:59 am

Sarepta shares buoyed by pledge to seek an early swift review for Duchenne gene therapy

Fierce Biotech

Fri, 07/29/22 - 10:20 am

Roche

accelerated approvals

Myocarditis case mars Sarepta DMD gene therapy readout

Pharmaforum

Thu, 07/7/22 - 10:23 am

myocarditis

Another Duchenne clinical hold gets lifted, this time at Dyne Therapeutics

Endpoints

Tue, 07/5/22 - 10:45 am

Dyne Therapeutics

DYNE-251

As muscular dystrophy drug meets key goal, Italfarmaco plans march forward to regulators

MedCity News

Mon, 06/27/22 - 10:15 am

Italy

Ifalfarmco

givinostat

FDA suspends US testing of Sarepta Duchenne drug over safety concerns

BioPharma Dive

Thu, 06/23/22 - 08:04 pm

SRP-5051

PTC Ready to Take Another Shot at FDA Approval of DMD Drug

BioSpace

Tue, 06/21/22 - 10:58 am

PTC Therapeutics

Translarna

Pfizer, Sarepta team with fellow DMD gene therapy makers to get to the bottom of adverse events

Fierce Biotech

Tue, 05/17/22 - 10:31 pm

With Sarepta in its sights, PepGen prices IPO to fund DMD trials

Fierce Biotech

Fri, 05/6/22 - 10:55 am

PepGen

IPOs

FDA clears Pfizer to restart Duchenne gene therapy trial, with new safeguards

BioPharma Dive

Thu, 04/28/22 - 10:56 am

clinical trial

Roche delays DMD drug deadline, decides to rejig MS trials because of Ukraine war

Fierce Biotech

Tue, 04/26/22 - 10:33 am

Roche

Ocrevus

Astellas takes $170m charge as it drops DMD gene therapies

Pharmaforum

Fri, 04/22/22 - 10:48 am

Astellas

earnings

Pfizer aims to restart late-stage trial of Duchenne gene therapy following safety setback

BioPharma Dive

Thu, 02/10/22 - 08:04 pm

Sarepta says early filing for DMD gene therapy may be back on

Pharmaforum

Wed, 01/12/22 - 10:53 am

Sarepta DMD Treatment Improves Motor Skills in Study Presented at JPM

BioSpace

Tue, 01/11/22 - 11:41 am

delandistrogene moxeparvovec

Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval

JPMHC 2022

BioPharma Dive

Mon, 01/10/22 - 11:39 pm

Pfizer reports patient death in early-stage Duchenne gene therapy trial, halts enrollment

Fierce Biotech

Tue, 12/21/21 - 10:28 am

patient deaths

PF-06939926

Buoyed by new data, Santhera preps filing for DMD drug vamorolone

Pharmaforum

Tue, 11/23/21 - 09:43 am

Santhera

DMD

idebenone

vamorolone

Pfizer tears up gene therapy timelines, revealing a series of phase 3 delays that end vision of a banner 2022

Fierce Biotech

Wed, 11/3/21 - 10:43 am

hemophilia