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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
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Crypto
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Read and Share Our Stories
Community Stories
Watch Family Videos
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For Researchers
Grants Overhead Policy
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See Our Fundraising Ideas
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Rare Disease Day
GM1 Gangliosidosis Awareness Day
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What We Do
About the Foundation
Mission Statement
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Informational Flyer
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Guide Families
Message to the Newly Diagnosed
Natural History Studies
Contact Us / Have questions?
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FDA Engagement, EL-PFDD
Voice of the Patient Report
Caregiver Preferences Study
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Research
2016 Cyclodextrin GM1 Pilot Study
By
raj
October 15, 2018
January 13, 2024
GM1 Cyclodextrin Pilot Study Initiated
This experiment was ceased due to lack of efficacy.
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2015-2017 Auburn/UMass Research Intravenous AAV9 Gene Therapy
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2017 UC Davis Lentiviral Gene Therapy Research
About GM1 Gangliosidosis
Toggle child menu
Expand
About
Toggle child menu
Expand
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
Toggle child menu
Expand
Advancements and Research
Gene Therapy
If Your Loved One Has GM1
Toggle child menu
Expand
Join a Natural History Study
Give Samples to the GM1 Biobank
Participate in Data Sharing
Contact Us
How to Help
Toggle child menu
Expand
Take Action
By Donating
Toggle child menu
Expand
Donate
Paypal Giving Fund
Crypto
Contact Us
Read and Share Our Stories
Toggle child menu
Expand
Community Stories
Watch Family Videos
Share Your Story
For Researchers
Toggle child menu
Expand
Grants Overhead Policy
By Fundraising
Toggle child menu
Expand
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
Toggle child menu
Expand
Rare Disease Day
GM1 Gangliosidosis Awareness Day
Move for GM1
Annual Conference
By Shopping
Toggle child menu
Expand
GM1 Store
RaiseRight
Buy Chocolate
eBay
Engage with Cure GM1
Toggle child menu
Expand
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
Toggle child menu
Expand
About the Foundation
Toggle child menu
Expand
Mission Statement
Board Members and Staff
Scientific and Business Advisors
Informational Flyer
Newsletters
Toggle child menu
Expand
View Newsletters
Subscribe to the Newsletter
Advancements / Research
Guide Families
Toggle child menu
Expand
Message to the Newly Diagnosed
Natural History Studies
Contact Us / Have questions?
Drive Progress and Research
Toggle child menu
Expand
FDA Engagement, EL-PFDD
Voice of the Patient Report
Caregiver Preferences Study