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Catching Up on PPMD’s 2026 Annual Conference: Part II

PPMD’s 2026 Annual Conference brought together individuals and families from across the Duchenne and Becker community for three days filled with scientific presentations, discussions around care, clinical trials and quality of life, and of course,…

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Catching up on What’s Happening at PPMD’S 2026 Annual Conference

PPMD’s 2026 Annual Conference is in full swing! PPMD’s 2026 Annual Conference kicked off with opening remarks from Katherine Beaverson, MD, CEO of PPMD, who welcomed attendees, introduced the dedicated PPMD team, recognized the many…

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Solid Biosciences Shares Updates on SGT-003

Solid Biosciences has shared a new letter to the Duchenne community providing updates on the development of SGT-003, the company’s investigational gene therapy for Duchenne. Solid shared that the ongoing INSPIRE DUCHENNE Phase 1/2 clinical…

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Capricor Therapeutics Shares Positive Five-Year HOPE-2 OLE Data for Deramiocel in Duchenne

Today, Capricor Therapeutics announced positive five-year data from the company’s ongoing HOPE-2 open label extension (OLE) study of Deramiocel, Capricor’s novel cell therapy, in individuals living with Duchenne. According to Capricor, among the nine patients…

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FDA Announces Advisory Committee Meeting to Review Capricor Therapeutics’ Biologics License Application for Deramiocel

The U.S. Food and Drug Administration (FDA) has scheduled an Advisory Committee meeting for July 29, 2026, to review Capricor Therapeutics’ Biologics License Application (BLA) for Deramiocel, an investigational cell therapy for the treatment of…

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Avidity Biosciences Submits Biologics License Application to FDA for del-zota

Today, Avidity Biosciences announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval of delpacibart zotadirsen (del-zota) for individuals living with Duchenne who are amenable…

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Inside the HHS Clinical Trials Roundtable: Why Speed, Access, and Rigor Matter for the Duchenne Community

On June 22, 2026, PPMD Chief Executive Officer Katherine Beaverson, MS attended the U.S. Department of Health and Human Services (HHS) Clinical Trials Roundtable in Washington, D.C., joining leaders from across government, industry, academia, and…

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Massachusetts Begins Newborn Screening for Duchenne

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…

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PPMD Joins FDA Leadership for Rare Disease Roundtable

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…

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Dyne Submits BLA to FDA for z-rostudirsen (DYNE-251) in Duchenne Amenable to Exon 51 Skipping

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…

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