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 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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October 11, 2024

WATCH: SHIELD DMD – A Bone Health Study in Duchenne (Webinar Recording)

Bone health is an important aspect of living with Duchenne muscular dystrophy. Roche recently joined PPMD for a webinar during which Maitea Guridi, a Principal Clinical Scientist at Roche, shared learnings around the natural history…

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October 9, 2024

Capricor Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002)

Capricor Therapeutics Initiates Rolling Submission of Biologics License Application (BLA) for Deramiocel (CAP-1002) for the Treatment of Duchenne Cardiomyopathy Capricor Therapeutics announced it has begun the rolling submission of its Biologics License Application (BLA) with…

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