Externally-Led Patient-Focused Drug Development Session On LGMD

• What will happen at this Externally-Led Patient Focus Drug Development (EL-PFDD) meeting? This important, monumental effort will give individuals diagnosed with limb-girdle muscular dystrophy types 2A, 2C, 2D, 2E, 2F, and 2I an opportunity to share with the FDA and other stakeholders about the experiences and challenges of living with LGMD.
• Where will the meeting take place? The meeting will take place virtually and will be live. Participants will be able to watch the event as it occurs.
• How can I get involved? You will be able to participate in surveys prior to the event to share your needs as an individual living with LGMD.  On the day of the meeting, we will hear several pre-recorded patient testimonies. You can also participate by answering live polling questions and by either calling or writing in with your comments. We want your voices to be heard!
• What do we hope to gain? The EL-PFDD meeting is designed to engage patients and elicit their unique perspectives. With this information, our goal is to enable knowledgeable development and review of LGMD therapies that meet the needs and expectations of our patient community. The meeting will be summarized in a Voice of the Patient report. The report, as well as recorded footage of the meeting, will be publicly available.

Please note that this initial endeavor represents Phase 1 and will include six LGMD subtypes. We anticipate additional LGMD subtypes may present to the FDA and other stakeholders at future EL-PFDD meetings.