The 2026 MDA Clinical & Scientific Conference Highlights Breakthroughs in Neuromuscular Research, Innovative Therapies, and Patient Care

The Muscular Dystrophy Association (MDA) concluded its 2026 MDA Clinical & Scientific Conference yesterday, convening over 2,400 attendees from 40 countries to help shape the future of neuromuscular ...

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 ...

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 ...
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 ...
BioSpace

BridgeBio Builds Case for Early 2027 Launch of Dystrophy Drug

Data from BridgeBio Pharma’s Phase 3 FORTIFY study show that BBP-418 significantly increases levels of a key disease biomarker that helps stabilize muscles in patients with limb-girdle muscular ...

First successes in the development of a gene therapy for incurable LAMA2-related muscular dystrophy

Researchers at the University of Basel have developed a gene therapy that could potentially treat a rare and currently fatal ...
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 ...
Stories by SWNS on MSN

Boy, 7, diagnosed with rare muscular condition after he stopped jumping

A little boy was diagnosed with a life-limiting, muscular condition after his parents realised he was unable to jump. Kairo ...
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 ...
Everyday Health

What Are the Causes of Muscular Dystrophy?

Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...

Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy

Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and ...
KETV Omaha

Omaha firefighters host steak fry to support families affected by muscular dystrophy

AFTER KETV NEWSWATCH SEVEN AT FOUR ON SUNDAY. IT IS AN EVENT THAT HAS BEEN GOING STRONG FOR OVER 70 YEARS. THIS YEAR, THE OMAHA FIRE DEPARTMENT’S STEAK FRY LOOKS TO BATTLE BACK AGAINST MUSCULAR ...