February 8, 2024

REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial

REGENXBIO Inc. has announced that enrollment has completed at dose level 2 of the Phase I/II AFFINITY DUCHENNE® trial of RGX-202, an investigational gene therapy product for the potential treatment of Duchenne. RGX-202 utilizes a…

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February 1, 2024

Duchenne Newborn Screening Bill Passes Arizona Senate

Earlier this week, Duchenne parent and PPMD Advocate Jill Castle testified before the Arizona State Senate Health & Human Services Committee in support of Senate Bill 1020, which seeks to add Duchenne to Arizona’s newborn…

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January 30, 2024

Watch: Navigating Steroid Choices – Understanding the Process, Pathways, and Policies (Webinar Recording)

Last week, PPMD was joined by Kathy Mathews, MD (Neurologist), Christina Trout, RN, MSN (Clinic Coordinator), and Rachel Kinn, PharmD, BCPPS (Pharmacist) from the University of Iowa to discuss decision making regarding steroids. In recent…

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January 29, 2024

PPMD 2023 Duchenne Early Care Meeting Report Published: Enhancing Support in Early Diagnosis

Last year, PPMD hosted a Duchenne Early Care meeting as part of the 2023 Duchenne Healthcare Professionals Summit, focusing on key aspects of care for the youngest children in our Duchenne community, in preparation for…

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January 29, 2024

Sarepta Therapeutics Reports Positive Data from Part B of MOMENTUM Study of SRP-5051

Sarepta Therapeutics, Inc. today announced positive data from Part B of SRP-5051-201, the MOMENTUM study. MOMENTUM is a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 that enrolled patients aged 8 to 21 years….

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January 26, 2024

Duchenne Added to Minnesota’s Newborn Screening Panel

PPMD is excited to announce another significant milestone: Minnesota has officially approved the addition of Duchenne to the state’s newborn screening panel! Approximately 63,000 babies are born in Minnesota every year, which means that we…

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January 25, 2024

CHMP Issues Negative Opinion for Renewal of Conditional Marketing Authorization for Translarna™ (ataluren) in Europe

PPMD is disappointed to learn that the European Medicines Agency (EMA)’s Committee for Medicinal Products for Human Use (CHMP) today issued a negative opinion following the re-examination procedure for the conditional marketing authorization of Translarna™…

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January 17, 2024

Astellas Releases Results of Discontinued Phase 1b Study of ASP0367

Astellas has shared results of the Phase 1b study of ASP0367 (MA-0211), an oral investigational drug. The study aimed to evaluate safety, tolerability, and preliminary efficacy in individuals living with Duchenne aged 8 to 16 years old….

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January 17, 2024

PPMD Hosts 2024 Duchenne Healthcare Professionals Summit

Each January, PPMD brings together experts in the field for our Duchenne Healthcare Professionals Summit to ensure that caring for individuals with the latest information and technology remains a constant priority.  Healthcare professionals from PPMD’s…

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January 11, 2024

Introducing PPMD’s Gene Therapy Hub

We are excited to announce the launch of PPMD’s Gene Therapy Hub! Gene therapy has made significant strides in Duchenne, with one therapy approved and other potential therapies in various stages of development. As our…

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