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 ...

Regenxbio reports Duchenne gene therapy progress as stock slides on mixed signals

Regenxbio Inc. RGNX shares are down on Wednesday after the company reported new interim data from its Phase 1/2 AFFINITY DUCHENNE trial of RGX-202. • Regenxbio stock is among today’s weakest ...

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 ...

Solid Biosciences Provides Interim Positive Clinical Update on Phase 1/2 INSPIRE DUCHENNE Trial

SGT-003 in the INSPIRE DUCHENNE trial to date - - Robust microdystrophin expression with consistent mechanistic evidence of dystrophin associated protein complex (DAPC) restoration and improvements in ...

Solid Bio falls amid new trial data for Duchenne drug

Solid Biosciences (SLDB) stock dips after new Phase 1/2 trial data for SGT-003, its gene therapy for Duchenne muscular dystrophy. Read more here, ...
BioSpace

Capricor Shares Rise as FDA Sets August Decision Date for Rejected Duchenne Therapy

Capricor Therapeutics’ deramiocel was rejected in July 2025, potentially caught between Nicole Verdun, a former top biologics regulator at the FDA, and Vinay Prasad, director of the Center for ...

Solid Biosciences Touts Encouraging Results For Duchenne Gene Therapy, FDA Talks Planned

Solid Biosciences Inc. SLDB shares are tumbling on Wednesday following a recent update regarding the company’s clinical trial for SGT-003, an investigational gene therapy. Disease-Relevant Treatment ...
BioSpace

Dyne Plans Post-Prasad FDA Run as Duchenne Exon Skipper Sustains Benefit in Long Term Data

Dyne Therapeutics is plotting an approval application for z-rostudirsen in the back half of 2026—a push that will only be bolstered by the departure of controversial CBER chief Vinay Prasad, according ...
People

Mom Has 3 Days to Get Son Life-Saving Treatment for Muscular Dystrophy Before He's Ineligible (Exclusive)

"My medicine is making me stronger," 7-year-old Hudson Sanford says after receiving a breakthrough gene therapy for Duchenne Muscular Dystrophy Cara Lynn Shultz is a writer-reporter at PEOPLE. Her ...

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 ...

The selected dose in the pivotal phase is lower than for other drug candidates in clinical trials or gene therapy drugs for Duchenne.

Parent Project Muscular Dystrophy, Foundation to Eradicate Duchenne Announce 2026 Advocacy Leadership Awards Recipients Honoring Bipartisan Champions in Congress

Parent Project Muscular Dystrophy (PPMD), in partnership with the Foundation to Eradicate Duchenne (FED), today announced Representative Troy Balderson (OH-12), Senator Susan Collins (ME), Senator Amy ...
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 ...