Gene Therapy for Duchenne Muscular Dystrophy: Genethon Confirms Two-Year Efficacy in Patients Treated with Its Drug Candidate GNT0004 at Therapeutic Dose in the First Phase of ...

Genethon, a pioneer and leader in gene therapy for rare diseases, unveiled results at the MDA Conference in Orlando confirming the long-term efficacy of its GNT0004 gene therapy in Duchenne muscular ...

Regenxbio's Duchenne gene therapy shows improved muscle function in trial

March 11 (Reuters) - Regenxbio said on Wednesday that interim data from a early-to-mid stage study of its experimental gene therapy in patients with Duchenne muscular dystrophy showed continued ...

Capricor Therapeutics Announces Late-Breaking HOPE-3 Data at the 2026 MDA Conference Demonstrating Significant Functional Benefits of Deramiocel for Duchenne Muscular Dystrophy

Reduced progression of myocardial fibrosis measured by LGE on cardiac MRI (p=0.022)Significant improvement in LVEF versus placebo in patients ...

BridgeBio Pharma eyes first treatment for rare muscle disorder as trial data shows early progress

BridgeBio Pharma Inc. BBIO on Wednesday announced a recent presentation of interim results from its Phase 3 FORTIFY trial for BBP-418, a treatment for limb-girdle muscular dystrophy type 2I/R9.
NC Biotech

Precision BioSciences cleared to begin gene therapy trial for muscular dystrophy

Durham-based Precision BioSciences, a gene-editing company, has received regulatory clearance to begin a clinical study of its potential treatment for Duchenne muscular dystrophy (DMD), a rare ...
KOBI-TV NBC5 / KOTI-TV NBC2

Part 2: Local boy with Duchenne Muscular Dystrophy

Seven-year-old William, a Southern Oregon boy living with Duchenne muscular dystrophy, is going to get a never-before-tried surgery designed specifically for his genetic mutation. NBC5’s medical ...
MedPage Today

Duchenne Muscular Dystrophy

While it’s established that Duchenne muscular dystrophy (DMD) causes a loss of muscle mass and strength, few studies have examined the gastrointestinal issues tied to DMD. Initial fractures are the ...
Science News

The first gene therapy for muscular dystrophy has been approved for some kids

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...
Hoodline

Ron Johnson Puts FDA On Hot Seat Over Pulled Muscular Dystrophy Drug

Ron Johnson is turning up the heat on the Food and Drug Administration after parents of boys with Duchenne muscular dystrophy told him the agency's recent decisions cut off access to a drug they ...
Your Observer

Island firefighters raise $3,600 for muscular dystrophy

Longboat Key firefighters “filled the boot” through a fundraising effort for the Muscular Dystrophy Association.
WebMD

Your Guide to Duvyzat for Duchenne Muscular Dystrophy

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...