February 6, 2023

Race Athlete Spotlight: Lauren Casey

It takes a village in a Duchenne/Becker Family.  Lauren Casey is an aunt to two boys living with Becker muscular dystrophy and has joined the Race to End Duchenne in their honor.  She took on…

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January 31, 2023

WATCH: Capricor Data Update From Ongoing HOPE-2 Open Label Extension Trial – Webinar Recording

WATCH: Capricor Data Update From Ongoing HOPE-2 Open Label Extension Trial Capricor Therapeutics recently joined PPMD for a community webinar on January 25, 2023 to present recent data findings from the ongoing HOPE-2 open label…

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January 26, 2023

PDC Submits White Paper Supporting Accelerated Approval for AAV Gene Therapies to FDA

PPMD is proud to contribute to a multi-stakeholder collaborative effort led by the Pathway Development Consortium (PDC) in the submission of a white paper to FDA on the use of the accelerated approval pathway for…

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January 25, 2023

PPMD Invests $500,000 in Myosana Therapeutics to Advance Non-Viral Gene Therapy Platform

Parent Project Muscular Dystrophy is excited to announce a $500,000 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company’s continuing development and translation of a non-viral gene therapy delivery platform aiming to slow…

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January 24, 2023

REGENXBIO Announces RGX-202 Gene Therapy Trial is Active & Recruiting

REGENXBIO has announced that the Phase I/II AFFINITY DUCHENNE™ trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne, is now active and recruiting trial participants. RGX-202 utilizes a novel adeno-associated virus (AAV8)…

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January 18, 2023

PPMD Urges FDA to Conduct a Full Review of Ataluren (Translarna)

Last month, PPMD held a webinar with PTC Therapeutics for a discussion regarding PTC’s history with the community and plans for the future, including activities to bring ataluren (Translarna) to US patients. Ataluren is an…

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January 12, 2023

Introducing PPMD Lighthouse Workshops: Let’s Navigate, Together.

Happy New Year friends. I hope this finds you greeting the start to 2023 rested and looking ahead with optimism and good energy. I am excited by many of the initiatives PPMD has in the…

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January 10, 2023

Your Registry Data at Work: More Complete Data Betters Our Understanding of Duchenne & Becker

In December, several team members of The Duchenne Registry attended the 2022 TREAT-NMD International Conference in Vancouver, Canada. The Registry has been a member of the TREAT-NMD neuromuscular network since we began collecting data in…

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January 10, 2023

Magical Miles & $300,000 Raised with the Race to End Duchenne at Walt Disney World Marathon Weekend

This past weekend, our team gathered at the most magical race weekend of the year – the Walt Disney World Marathon Weekend, presented by State Farm. And THIS is the magic of us! Our Race…

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January 9, 2023

Update from Santhera & ReveraGen: FDA Acceptance of New Drug Application for Vamorolone in Duchenne

PPMD is excited to learn from Santhera and ReveraGen that the FDA has accepted the New Drug Application (NDA) for vamorolone, an investigational dissociative steroid for individuals with Duchenne. Under the Prescription Drug User Fee…

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