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Research

The Voice of the Patient Report from the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development Meeting (EL-PFDD)

Byraj February 23, 2023November 14, 2023

You can now download and read the touching and impactful summary of input from GM1 gangliosidosis caregivers and family given during the October 2022 GM1 Externally-led patient-focused drug development meeting with the FDA (EL-PFDD).

The GM1 Gangliosidosis Voice of the Patient Report

You can also view the report on the FDA’s website.

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About the GM1 Gangliosidosis Externally-Led Patient-Focused Drug Development (EL-PFDD) Meeting with FDA
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  • About GM1 Gangliosidosis
    • About
      • About GM1
      • Disease Overview PDF
      • Clinical Disease Overview Video
      • The Cause
      • Who Gets GM1
      • Incidence of GM1
      • History of GM1
      • Life Expectancy
      • Subtypes of GM1
    • Treatments and Research
      • Advancements and Research
    • If Your Loved One Has GM1
      • Join a Natural History Study
      • Give Samples to the GM1 Biobank
      • Participate in Data Sharing
      • Contact Us
  • How to Help
    • Take Action
    • By Donating
      • Donate
      • Paypal Giving Fund
      • Crypto
      • Contact Us
    • Read and Share Our Stories
      • Community Stories
      • Watch Family Videos
      • Share Your Story
    • For Researchers
      • Grants Overhead Policy
    • By Fundraising
      • Fundraise for CureGM1
      • Use Our Fundraising Manual
      • Fundraise with Charity Miles App
      • See Our Fundraising Ideas
      • Used Car Donation
      • GoFundMe
      • JustGiving
      • Facebook
    • Participate in Our Events
      • Rare Disease Day
      • GM1 Gangliosidosis Awareness Day
      • Move for GM1
      • Annual Conference
    • By Shopping
      • GM1 Store
      • RaiseRight
      • Buy Chocolate
      • eBay
    • Engage with Cure GM1
      • Contact Us
      • Subscribe to the Newsletter
      • Join the Cure GM1 Parents Facebook Group
      • Join the Friends of Cure GM1 Facebook Group
    • Subscribe to Our YouTube Channel
  • What We Do
    • About the Foundation
      • Mission Statement
      • Board Members and Staff
      • Scientific and Business Advisors
      • Informational Flyer
    • Newsletters
      • View Newsletters
      • Subscribe to the Newsletter
    • Advancements / Research
      • Enzyme Replacement Therapy Project
    • Guide Families
      • Message to the Newly Diagnosed
      • Learn about Data Sharing
      • Contact Us / Have questions?
    • Drive Progress and Research
      • FDA Engagement, EL-PFDD
      • Voice of the Patient Report
      • Caregiver Preferences Study