WE NEED YOU!

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Join us September 23, 2022, when we meet with the FDA and other stakeholders about our needs as a patient community!

The goal of this meeting is to provide the U.S. Food and Drug Administration (FDA), product developers, clinicians, industry, and academic researchers an opportunity to hear perspectives from individuals with LGMD on the health effects, daily impacts, treatment goals, and decision factors considered when seeking out or selecting a treatment. This meeting is being conducted as a parallel effort to FDA’s EL-PFDD initiative, a commitment under the fifth authorization of the Prescription Drug User Fee Act (PDUFA V) to more systematically gather patients’ perspectives on their conditions and available therapies to treat their conditions.

• What will happen at this 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 also be able to participate in polling and 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 provide information to help the FDA review new drug applications for LGMD therapies to try and best 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 will pursue EL-PFDD meetings in the future.