November 20, 2024

PPMD Culminates 30th Anniversary with Celebratory “Investing in Every Future” Event

PPMD commemorated its 30th anniversary with the “Investing in Every Future” celebratory event on November 14, 2024, in New York City. With some 200 attendees, this meaningful gathering honored three decades of progress and the resilience…

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November 18, 2024

REGENXBIO Initiates Pivotal Phase of AFFINITY DUCHENNE® Trial of RGX-202 Gene Therapy, Reports Positive Functional Data

REGENXBIO Inc. has announced that the AFFINITY DUCHENNE® open-label trial of RGX-202 has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data from the Phase…

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November 12, 2024

Calling All Adults with Duchenne & Becker: Join the 2025 PPMD Adult Advisory Committee

Have you been looking for a way to engage with the community more? Do you have a personal experience that you would like to share with the larger community or through federal and state advocacy…

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November 7, 2024

REGENXBIO Shares Updates on Phase I/II AFFINITY DUCHENNE® trial of RGX-202

Yesterday, REGENXBIO shared important updates on their Phase I/II AFFINITY DUCHENNE® trial of RGX-202 for Duchenne during their third quarter financial results update. RGX-202 utilizes a novel adeno-associated virus (AAV8) to transport a shortened version…

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November 7, 2024

Solid Biosciences Shares Updates on INSPIRE DUCHENNE Clinical Trial of SGT-003

Yesterday, Solid Biosciences shared new updates on the INSPIRE DUCHENNE clinical trial for their next-generation AAV micro-dystrophin gene therapy candidate, SGT-003, for the treatment of Duchenne. Based on early study results, the trial has expanded…

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November 6, 2024

Sarepta Announces Discontinuation of SRP-5051 Development for Duchenne

Sarepta Therapeutics today announced that the company is discontinuing its SRP-5051 (vesleteplirsen) development program, including the global, Phase 2, multi-arm, ascending dose MOMENTUM study. Vesleteplirsen is an investigational, next-generation treatment utilizing Sarepta’s PPMO chemistry and…

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November 5, 2024

WATCH: Exploring Muscle Regeneration – A New Approach with Satellos (Webinar Recording)

Satellos recently joined PPMD for a community webinar exploring a new approach for the treatment of Duchenne. Satellos presented on the discovery behind SAT-3247, a small molecule drug that mobilizes the body’s own muscle stem…

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October 30, 2024

FDA Accepts Translarna NDA Resubmission for Review

A Letter From PPMD’s Pat Furlong I am thrilled to share that the FDA has officially accepted PTC Therapeutics’ New Drug Application (NDA) resubmission for Translarna (ataluren). This acceptance is an important step, meaning the…

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October 29, 2024

Flu Season Ready: Protecting Yourself and Your Family

With the start of school and the onset of winter, we can expect a rise in respiratory viruses. This year, it’s crucial to safeguard yourself, your children, and your family from all respiratory infections, particularly…

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October 16, 2024

Catch Up on World Muscle Society 2024

Each year, the World Muscle Society (WMS) Annual Congress brings together the best minds in neuromuscular research, clinical care, patient advocacy, and industry. It offers a dynamic platform for sharing new data, fostering collaborations, and…

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