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PPMD’s 2020 Virtual Annual Conference Agenda – Now Available!

PPMD’s 2020 Virtual Annual Conference Agenda – Now Available! PPMD is excited to unveil our full agenda for the 2020 Virtual #PPMDConference, July 22-25! This summer continues to be an unprecedented time for our global community,…

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Applications Open for PPMD and Duchenne UK $1 Million Joint Research Grant Call for Gene Therapy

PPMD is thrilled to announce our partnership with Duchenne UK to launch a joint 2020 Research Grant Call, inviting research proposals for submission and review. Up to $1 million (US) is committed to a project…

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WATCH: Family Friday – When You’re a Carrier

Family Friday: When You’re a Carrier Learning that you are a female carrier of Duchenne can be shocking, overwhelming, and confusing. During our June 26, 2020 Family Friday on Facebook Live, we were joined by…

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End Duchenne Tour 2020: Virtual Tour Stop in Long Island

End Duchenne Tour 2020: Virtual Tour Stop in Long Island On the morning of May 30, PPMD hosted our 4th End Duchenne Tour Stop of 2020. This gathering marked our 3rd iteration of our virtual…

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Sarepta Completes Submission of New Drug Application Seeking Approval of Casimersen (SRP-4045)

PPMD is excited to learn that Sarepta Therapeutics has completed the submission of a rolling New Drug Application (NDA) to the FDA seeking accelerated approval for casimersen (SRP-4045). Casimersen, a phosphorodiamidate morpholino oligomer (PMO), is…

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WATCH: Family Friday – Navigating Your Neuromuscular Team

Family Friday: Navigating Your Neuromuscular Team For many of us, self-quarantine is in full swing, and adjusting to a different family structure may be overwhelming. So your PPMD family is launching Family Fridays to discuss…

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WATCH: Antisense Therapeutics Phase II Trial Final Data Results – Webinar Recording

Antisense Therapeutics recently announced that their Phase II clinical trial of ANP’s immunomodulatory therapy, ATL1102 for Duchenne in non-ambulatory patients has met its primary endpoint, confirming the safety and tolerability of ATL1102 for advancement into…

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Download PPMD’s New Care Checklists

PPMD knows that keeping track of all elements of your/your child’s care can be overwhelming and confusing. For this reason, we have created a series of care checklists for families to track and understand recommended…

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Sarepta Announces Positive Safety and Efficacy Data from SRP-9001 Gene Therapy Trial Published

Sarepta Therapeutics today announced safety and tolerability data at one year from four Duchenne clinical trial participants who received SRP-9001 micro-dystrophin (AAVrh74.MHCK7.micro-dystrophin) have been published in JAMA Neurology. PPMD is proud to have been an…

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WATCH: Family Friday – Advocating for Yourself in Research

Family Friday: Reflecting on Adjusting to a New Diagnosis For many of us, self-quarantine is in full swing, and adjusting to a different family structure may be overwhelming. So your PPMD family is launching Family…

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