Kids News

As of today, Massachusetts has officially begun screening all newborns for Duchenne muscular dystrophy. This milestone comes after the passage of the state’s Maternal Health bill (H. 4999), which was signed into law by Governor… The post Massachusetts Begins Newborn Screening for Duchenne appeared first on Parent Project Muscular Dystrophy.

On Wednesday, June 3rd, PPMD was invited to participate in a closed roundtable with senior leadership at the U.S. Food and Drug Administration (FDA). The meeting was convened by Amy Comstock Rick, JD, Director of… The post PPMD Joins FDA Leadership for Rare Disease Roundtable appeared first on Parent Project Muscular Dystrophy.

Today, Dyne Therapeutics announced that the company has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, DYNE-251) for individuals living with Duchenne who are amenable… The post Dyne Submits BLA to FDA for z-rostudirsen (DYNE-251) in Duchenne Amenable to Exon 51 Skipping appeared first on Parent Project Muscular Dystrophy.

Dyne Therapeutics today announced the initiation of the company’s Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, DYNE-251), in individuals living with Duchenne who are amenable to exon 51 skipping. Z-rostudiresen is an investigational exon… The post Dyne Announces Phase 3 Trial of Z-Rostudirsen, Plans for Submitting Biologics License Application to FDA appeared first on Parent Project Muscular Dystrophy.

What can you learn from the PPMD Adult Advisory Committee (PAAC) that you can’t ask Google? Honestly? A lot. Google can give you information, but it can’t tell you what it’s actually like to live with… The post NEW: Livestream with the PAAC appeared first on Parent Project Muscular Dystrophy.