Kids News

Today PTC Therapeutics released an updated letter to the Duchenne community on their website with additional details following the company’s withdrawal of the ataluren New Drug Application (NDA). This update includes new information on a… The post PTC Therapeutics Shares Updated Community Letter Regarding Ataluren appeared first on Parent Project Muscular Dystrophy.

PPMD and Secretome Therapeutics (Secretome) are excited to announce that PPMD has provided $250,000 in funding to Secretome through PPMD Venture Pathways, our venture-philanthropy initiative that provides funding to industry to accelerate therapeutic development for… The post PPMD Provides $250,000 in Funding to Secretome Therapeutics Through PPMD Venture Pathways Program to Support Development of Cardiac Cell Therapy appeared first on Parent Project Muscular Dystrophy.

Today, Sarepta Therapeutics announced plans to submit supplemental New Drug Applications (sNDAs) to the U.S. Food and Drug Administration (FDA) for AMONDYS 45 and VYONDYS 53, the company’s exon-skipping therapies for the treatment of individuals… The post Sarepta Announces Plans to Submit sNDAs for AMONDYS 45 and VYONDYS 53 appeared first on Parent Project Muscular Dystrophy.

Sarepta Therapeutics, Inc. has announced screening and enrollment are underway in Cohort 8 of the company’s ENDEAVOR study. ENDEAVOR is an open-label, Phase 1b study assessing the expression and safety of ELEVIDYS in multiple cohorts… The post Sarepta Announces Initiation of Screening and Enrollment for ENDEAVOR Cohort 8 in Non-Ambulatory Individuals Living with Duchenne appeared first on Parent Project Muscular Dystrophy.

Each year, the Duchenne and Becker muscular dystrophy community comes together in Washington, DC with a shared mission: to ensure that the voices of individuals living with Duchenne and Becker and their families are heard… The post Community Comes Together on Capitol Hill: A Recap of PPMD’s 2026 Advocacy Conference appeared first on Parent Project Muscular Dystrophy.