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Home / Blog - Page 16

Blog

2017 Dorphan Small Molecule Research and Mouse Study

October 17, 2018November 27, 2023

Cure GM1 granted funds for a drug repurposing project and mouse study to study a…

Read More 2017 Dorphan Small Molecule Research and Mouse StudyContinue

2017 UC Davis Lentiviral Gene Therapy Research

October 16, 2018November 27, 2023

Cure GM1 recently granted funds towards the development of a lentiviral gene therapy approach using…

Read More 2017 UC Davis Lentiviral Gene Therapy ResearchContinue

2016 Cyclodextrin GM1 Pilot Study

October 15, 2018January 13, 2024

GM1 Cyclodextrin Pilot Study Initiated This experiment was ceased due to lack of efficacy.

Read More 2016 Cyclodextrin GM1 Pilot StudyContinue

2015-2017 Auburn/UMass Research Intravenous AAV9 Gene Therapy

October 14, 2018January 13, 2024

Cure GM1 funded pre-clinical studies, IND-enabling studies, and the Pre-IND briefing document for intravenous AAV9…

Read More 2015-2017 Auburn/UMass Research Intravenous AAV9 Gene TherapyContinue

Syner-G Clinical Study

October 13, 2018November 27, 2023

This clinical study was being conducted at University of Minnesota where the ketogenic diet was…

Read More Syner-G Clinical StudyContinue

Althea’s Story

July 19, 2018November 27, 2023

Althea was born on 23rd September 2016, a beautiful 6lb 8oz baby girl. Thea was…

Read More Althea’s StoryContinue

Paxton’s Story

February 28, 2018November 27, 2023

A beautiful baby boy born February 16, 2012 Paxton is our first and only child,…

Read More Paxton’s StoryContinue

Kate’s Story #AWin4Kate

February 28, 2018November 27, 2023

#AWin4Kate Kate was born May 17, 2011, a healthy and happy baby. We always thought…

Read More Kate’s Story #AWin4KateContinue

Joe’s Story

February 28, 2018November 27, 2023

A special boy that fought to the end Joe was born 4 weeks early by…

Read More Joe’s StoryContinue

Paola’s Story

February 28, 2018November 27, 2023

Una Bellisima Bambina Paola is a beautiful child born on June 29, 2013. She was…

Read More Paola’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
    • 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