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Joaquim and Sofia’s Story

October 18, 2024October 18, 2024

Joaquim and Sofia’s story, by their father “Our current focus is on keeping Joaquim as…

Read More Joaquim and Sofia’s StoryContinue

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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
      • Gene Therapy
    • 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
    • Guide Families
      • Message to the Newly Diagnosed
      • Natural History Studies
      • Contact Us / Have questions?
    • Drive Progress and Research
      • FDA Engagement, EL-PFDD
      • Voice of the Patient Report
      • Caregiver Preferences Study