Aug 22, 2024 · Hiram Secrist, the first boy treated with Elevidys gene therapy following FDA approval, has seen significant improvements in the first year.

Jun 17, 2024 · Exercise and physical therapy can help a person with Duchenne muscular dystrophy maintain mobility, flexibility, and independence.

Sep 12, 2024 · The U.S. Food and Drug Administration granted rare pediatric disease designation to NS-050/NCNP-03, an exon-skipping DMD treatment.

Oct 15, 2024 · A single low dose of the investigational gene therapy BB-301 helped with swallowing for the first two people with oculopharyngeal muscular dystroph y (OPMD) treated in a Phase 1b/2a clinical trial, according to an update from the …

Jun 21, 2024 · Assistive technology for Duchenne muscular dystrophy includes adaptive aids and modifications that help with independent living.

Jan 2, 2025 · In 2024, Muscular Dystrophy News Today covered the latest studies, treatment advances, and clinical trials for different types of the disease.

Nov 21, 2024 · Regenxbio has initiated the pivotal phase of a clinical trial testing its experimental gene therapy RGX-202 in boys with Duchenne muscular dystrophy (DMD).

Oct 10, 2024 · Capricor Therapeutics started its rolling submission seeking FDA approval of its DMD therapy deramiocel for heart muscle disease in patients.

Dec 3, 2024 · Sarepta has acquired the global rights to develop ARO-DUX4 and ARO-DM1, two RNAi therapeutic candidates for types of muscular dystrophy.

Mar 14, 2024 · The investigational cell therapy CAP-1002 has continued to slow declines in upper limb and heart function among boys and young men with Duchenne muscular dystrophy (DMD), according to two-year results from the HOPE-2 open label extension (OLE) study.