October 14, 2023

WATCH: Affinity Duchenne™ and Affinity Beyond™: Updates on Investigational RGX-202 from REGENXBIO (Webinar Recording)

REGENXBIO joined PPMD for a community webinar on October 11, 2023 to discuss the company’s investigational gene therapy for Duchenne muscular dystrophy, AFFINITY DUCHENNE™, and AFFINITY BEYOND™, an AAV8 antibody assessment study. Jahannaz (Naz) Dastgir,…

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September 29, 2023

WATCH: Advocating for Access: Insurance 101 (Webinar Recording)

Navigating insurance can be daunting – from decoding acronyms, to understanding the types of health plans, and more. During September’s Advocating for Access webinar, the PPMD and Little Hercules Foundation teams dug into some of…

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August 25, 2023

WATCH: Advocating for Access: Navigating Approved Therapies in Duchenne (Webinar Recording)

Understanding and navigating insurance access can be daunting. That’s why PPMD kicked off a new webinar series, “Advocating for Access,” earlier this week with its first monthly installment on Navigating Approved Therapies in Duchenne. PPMD’s…

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July 14, 2023

WATCH: ELEVIDYS: What This Means for You and Your Family (Webinar Recording)

In case you missed it, PPMD was joined by Drs. Russ Butterfield of University of Utah and Sue Apkon of Colorado Children’s earlier this week to talk through some of the common questions around the…

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June 21, 2023

WATCH: ENVISION Study Update With Sarepta Therapeutics (Webinar Recording)

Last week Sarepta Therapeutics joined PPMD to discuss ENVISION, their upcoming 18-month placebo-controlled Phase 3 study of SRP-9001 (delandistrogene moxeparvovec) for older ambulatory and non-ambulatory individuals with Duchenne. Matthew Furgerson, PhD, Medical Director, Clinical Development…

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May 19, 2023

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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May 11, 2023

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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April 20, 2023

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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April 15, 2023

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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March 27, 2023

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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