Blog

SRP-9001: Submit Your Community Perspectives to FDA

On May 12, the FDA held an Advisory Committee meeting for SRP-9001, a gene therapy intended to treat Duchenne. The Advisory Committee meeting was used by FDA to obtain independent expert advice on scientific, technical,…

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WATCH: Edgewise — A Novel Approach Designed to Protect Muscle in Becker (Webinar Recording)

PPMD was joined by Edgewise Therapeutics for a community webinar on May 3, 2023 to discuss the company’s novel approach designed to protect muscle in individuals living with Becker muscular dystrophy. The webinar highlighted EDG-5506,…

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WATCH: Dyne Therapeutics — Advancing FORCE in the Pursuit of Therapies (Webinar Recording)

Dyne Therapeutics joined PPMD for a community webinar on April 12, 2023 to discuss Dyne’s FORCE™ platform. Ashish Dugar, PhD, MBA, Dyne’s Chief Medical Affairs Officer, and Molly White, Dyne’s Vice President, Global Head of…

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WATCH: Connect With the Experts – Bone Health in Duchenne (Webinar Recording)

Connect With the Experts – Bone Health in Duchenne PPMD recently hosted a webinar led by expert endocrinologists Dr. Leanne Ward (University of Ottawa), Dr. Janet Crane (Johns Hopkins) and Nadia Merchant (Children’s National) to…

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WATCH: Viltepso, An FDA-Approved Treatment for Patients Amenable to Exon 53 Skipping – Webinar Recording

PPMD and NS Pharma hosted a community webinar on March 2, 2023 to discuss VILTEPSO, an FDA-approved treatment for patients with Duchenne amenable to exon 53 skipping. Mathula Thangarajh, MD joined us to discuss the…

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WATCH: PPMD, NORD and PQHE Present ‘Living with Duchenne Muscular Dystrophy in 2023’

PPMD, NORD and PQHE Present ‘Living with Duchenne Muscular Dystrophy in 2023’ PPMD and the National Organization for Rare Disorders (NORD) recently hosted a community webinar discussing the current and future landscape of Duchenne care…

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February 13, 2023

Watch: Avidity Biosciences – EXPLORE44 Clinical Trial Investigating AOC 1044 – Webinar Recording

WATCH: Avidity Biosciences – EXPLORE44 Clinical Trial Investigating AOC 1044 in Individuals Amenable to Exon 44 Skipping Avidity Biosciences recently joined PPMD for a community webinar on February 8, 2023 to discuss the EXPLORE44™ clinical…

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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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December 21, 2022

WATCH: Protect Your Lungs This Winter (Webinar Recording)

Connect With the Experts – Pulmonary Management in Winter Season As we embark into the winter season, it’s imperative that individuals with Duchenne and Becker muscular dystrophy are prepared to manage their pulmonary health and…

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December 19, 2022

WATCH: A Conversation with PTC Therapeutics (Webinar Recording)

WATCH: A Conversation with PTC Therapeutics (Webinar Recording) PTC Therapeutics recently joined PPMD for a community webinar on Friday, December 9, 2022 for a discussion regarding PTC’s history with the community and plans for the…

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