Kids News

Solid Biosciences Inc. has shared that the first participant has been dosed in IMPACT DUCHENNE, the company’s multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003 for the treatment of Duchenne muscular dystrophy. SGT-003… The post Solid Biosciences Doses First Participant in Phase 3 IMPACT DUCHENNE Clinical Trial Evaluating SGT-003 in Duchenne appeared first on Parent Project Muscular Dystrophy.

Duchenne was added to the Recommended Uniform Screening Panel (RUSP) (RUSP) on December 16, 2025, and in the months since, that decision, coupled with our continued advocacy efforts, have driven meaningful action at the state… The post Momentum Continues: Duchenne Newborn Screening Advances Across the US appeared first on Parent Project Muscular Dystrophy.

Entrada Therapeutics has announced positive topline data from Cohort 1 of the double-blind, placebo-controlled, multiple ascending dose portion of the Phase 1/2 ELEVATE-44-201 study evaluating ENTR-601-44 in ambulatory participants ages 4–20 living with Duchenne muscular… The post Entrada Reports Positive Topline Results from Phase 1/2 ELEVATE-44-201 Study in Duchenne appeared first on Parent Project Muscular Dystrophy.

This past weekend, PPMD kicked off our 2026 PPMD Together series in Philadelphia, Pennsylvania. Individuals with Duchenne and Becker, their families, clinicians, and industry partners gathered for a day and a half full of connection… The post Catch up on PPMD Together: Philadelphia appeared first on Parent Project Muscular Dystrophy.

On April 24, 2026, PPMD, in collaboration with the Muscular Dystrophy Association (MDA) and the Little Hercules Foundation (LHF), convened a Duchenne Newborn Screening (NBS) Stakeholder Meeting in Philadelphia. The meeting’s main goal was to… The post Advancing Duchenne Newborn Screening: A Collaborative Path Forward appeared first on Parent Project Muscular Dystrophy.