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Join us September 23rd, 2022 as we meet with the FDA and other stakeholders about our needs as an LGMD community!

  • 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 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. Many individuals will be selected to speak to the FDA and other stakeholders either through prerecorded video or during live segments in the EL-PFDD.
  • 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. Many individuals will be selected to speak to the FDA and other stakeholders either through prerecorded video or during live segments in the EL-PFDD.
  • 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 the FDA and the whole drug development community to understand the needs of our patient community and our expectations for future treatments. 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 present to the FDA and other stakeholders at future PFDD meetings.

WE NEED YOU!

Do you want to help us make historical changes for the limb-girdle community?

If so, we need your voice! The subtypes of 2A, 2C, 2D, 2E, 2F, and 2I will be highlighted in this first phase of the PFDD meeting.

Learn more at https://thespeakfoundation.com/pfdd

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