November 6, 2023

WATCH: AGAMREE Approval – What this means for you & your family (Webinar recording)

PPMD was recently joined by Dr. Aravindhan Veerapandiyan of Arkansas Children’s Hospital to discuss the FDA’s recent approval of AGAMREE (vamorolone), a new steroid approved for the treatment of Duchenne muscular dystrophy in individuals aged…

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October 31, 2023

WATCH: Advocating for Access – Overview of Medicaid DUR and P&T Meetings (Webinar Recording)

During October’s Advocating for Access webinar, the PPMD and Little Hercules Foundation teams dug into what happens after a drug is approved with regard to how state Drug Utilization Review (DUR) boards assess how a…

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October 30, 2023

WATCH: Capricor Therapeutics – CAP-1002 HOPE-3 Program Update (Webinar Recording)

PPMD and Capricor Therapeutics held a community webinar on October 25, 2023 to provide an overview of the latest regulatory developments for Capricor’s ongoing HOPE-3 Phase 3 study of CAP-1002, the company’s cell therapy. Capricor’s…

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October 24, 2023

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

Edgewise Therapeutics joined PPMD for a community webinar on October 18, 2023 to discuss the company’s investigational compound, EDG-5506. Joanne M. Donovan, M.D., Ph.D., Chief Medical Officer at Edgewise, and Alan Russell, Ph.D., Edgewise’s Co-Founder and…

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October 17, 2023

WATCH: Supporting Families Through Decision Making (Webinar Recording)

PPMD webinar explores how palliative care teams can support families through decision making PPMD’s Alexis Hazlett and Patrick Moeschen were recently joined by panelists Dr. Lauren Treat, Neurologist and Palliative Care Physician at Colorado Children’s,…

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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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