CTTI Project: Patient Group Engagement
Webinar Presenters:
- Sharon Hesterlee, Ph.D., Vice President Research, Parent Project Muscular Dystrophy
- Holly Peay, MS CGC, Vice President, Education and Outreach, Parent Project Muscular Dystrophy, and Director, DuchenneConnect Registry
*The views and opinions expressed in this video are those of the speaker and do not necessarily reflect the official policy or position of CTTI.
Webinar Objective
Sharon Hesterlee and Holly Peay discuss the ways in which Parent Project Muscular Dystrophy (PPMD) has partnered with industry to accelerate therapy development for Duchenne Muscular Dystrophy.
Webinar Resources:
- FDA Guidance on Duchenne
- Putting Patients First: Speeding Responsible Access (PPMD White Paper)
- Benefit-Risk Assessments in Rare Disorders (PPMD White Paper)
- A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy (Clinical Trials, Volume 36, Issue 5)
- PPMD Patient Registry