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 ...

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.
BioSpace

MDA 2026: Biogen, Sarepta, Edgewise Address Muscle Disorders

Data presented at the 2026 Muscular Dystrophy Association meeting could have readthroughs to companies developing therapies ...

Satellos Presents Interim SAT-3247 Clinical and Biomarker Data in Duchenne Muscular Dystrophy at the 2026 MDA Clinical & Scientific Conference

Interim observations from the Phase 2 TRAILHEAD study show continued improvement in handgrip strength, overall stability of elbow and shoulder ...

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 with baseline cardiomyopathy (p=0.017)Global Statistical ...

Edgewise Announces Positive Long-Term Sevasemten Data Demonstrating Sustained Functional Stabilization in Becker Muscular Dystrophy Through 3.5 Years of Follow-Up

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today presented at the 2026 MDA Clinical and Scientific Conference, long-term data from its MESA ...
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 ...

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 ...
TMCnet

Tenaya Therapeutics Presents Preclinical Data at MDA 2026 Highlighting TN-301's Potential to Correct Skeletal and Cardiac Muscle Decline in Duchenne Muscular Dystrophy

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of ...
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 ...

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 ...