Duchenne Muscular Dystrophy

Biogen

Vertex flexes its dealmaking muscle

EP Vantage

Mon, 03/27/23 - 10:07 am

Vertex Pharmaceuticals

Entrada Therapeutics

Tevard Biosciences

FDA Reverses Course; Will Hold Adcomm for Sarepta’s DMD Gene Therapy

BioSpace

Fri, 03/17/23 - 10:13 am

Duchenne muscular dystrophy: major trials and events to watch in 2023

Clinical Trials Arena

Thu, 03/16/23 - 09:49 am

Sarepta: FDA finds ‘no significant safety issues of concern’ with DMD gene therapy

Biopharma Reporter

Mon, 03/6/23 - 11:29 pm

Sarepta's DMD gene therapy inches closer to market as FDA declines to hold advisory meeting

Fierce Biotech

Wed, 03/1/23 - 09:52 am

Vertex tiptoes further into Duchenne research with Tevard deal

BioPharma Dive

Tue, 02/28/23 - 10:37 pm

Vertex Pharmaceuticals

Tevard Biosciences

Duchenne muscular dystrophy’s progress so far in 2023

Clinical Trials Arena

Wed, 02/22/23 - 10:00 am

Santhera Pharmaceuticals

FDA accepts Santhera, ReveraGen’s vamorolone NDA to treat DMD

Pharmaceutical Business Review

Tue, 01/10/23 - 10:42 am

Santhera Pharmaceuticals

ReveraGen

vamorolone

Sarepta marches forward with its potential DMD gene therapy and an expanded Catalent manufacturing deal

Endpoints

Thu, 01/5/23 - 04:58 pm

CDMOs

Catalent

Fresh from Vertex payday, Entrada's DMD plans are derailed by FDA hold

Fierce Biotech

Tue, 12/20/22 - 10:42 am

Entrada Therapeutics

Vertex Pharmaceuticals

clinical trials

myotonic muscular dystrophy

ENTR-601-44

FDA grants speedy review to Sarepta’s Duchenne gene therapy

BioPharma Dive

Tue, 11/29/22 - 10:50 am

FDA preps for DMD drug generics as Sarepta has yet to finish its confirmatory trial

Endpoints

Sat, 11/26/22 - 07:15 pm

generics

eteplirsen

Only Patient in N-of-1 CRISPR Trial for Duchenne Muscular Dystrophy Dies

BioSpace

Mon, 11/7/22 - 10:45 am

CRISPR

clinical trials

patient deaths

CRD-TMH-001

Sarepta preps $1.2B war chest as DMD gene therapy nears accelerated FDA nod

Fierce Biotech

Thu, 11/3/22 - 11:56 am

Santhera seeks speedy FDA review of Duchenne drug vamorolone

Pharmaphorum

Fri, 10/28/22 - 10:26 am

Santhera

vamorolone

Sarepta asks FDA to approve first gene therapy for Duchenne muscular dystrophy

BioPharma Dive

Thu, 09/29/22 - 07:38 pm

Pepgen aims to overtake Sarepta

EP Vantage

Wed, 09/28/22 - 11:00 am

PepGen

Eyeing accelerated approval for Duchenne gene therapy, Sarepta hopes to bring in $1B in bid for profitability

PGN-ED051

Endpoints

Tue, 09/13/22 - 10:35 am

After serious side effect, Sarepta and FDA realign on next-gen Duchenne drug with trial hold lifted

Endpoints

Tue, 09/6/22 - 10:48 am