Join Cure GM1โs initiative to support natural history data collection.
A Patient-Led Nonprofit Effort
Cure GM1 Foundation has initiated data collection through the Matrix platform to collect natural history data and real-world data to support the development of possible treatments for GM1 gangliosidosis. Families can participate in these efforts to ensure that GM1 gangliosidosis is research-ready!
This GM1-specific platform will allow us to create a truly robust dataset. This patient portal and registry is a significant investment and step forward. We are pleased to be a member of COMBINEDBrain, which leverages the Matrix platform for this purpose. The data collected is critically important to support the drug development process.
Every Family Matters in GM1 Research
Research is the foundation for finding treatments and a cure for GM1 gangliosidosis, but it cannot happen without YOU.
With a disease as rare as GM1 gangliosidosis, every family who participates brings us one step closer to breakthroughs. Your story creates the roadmap scientists need to develop new therapies.
Why Your Participation Matters
Show We Exist
Help healthcare providers, researchers, and pharmaceutical companies understand GM1 gangliosidosis and our community
Drive Progress
Your data and samples enable breakthrough discoveries and clinical trials
Privacy Protected
All published data is anonymized and used solely to advance research.
Spread Awareness and Make GM1 visible
The LabCorp Patient Insights Network dataset contributed to this dataset from 2019-2024 and includes 165 patients.
What is the GM1 Matrix
The GM1 Matrix is a comprehensive research study conducted by the Cure GM1 Foundation and COMBINEDBrain to collect information from patients and families affected by GM1 gangliosidosis worldwide. The GM1 Matrix collects data, including GM1 subtypes, diagnostic journeys, symptoms, impacts of GM1 gangliosidosis on daily life, and medical records.
Who can participate?
- People affected by GM1 gangliosidosis who are legal adults in their country of residence
- Parents of a child with a diagnosis of GM1 gangliosidosis that was confirmed by a doctor or another healthcare professional
- Legal guardians or authorized representatives of adults with GM1 gangliosidosis
Participants of all ages are eligible for this study, but the person completing the survey must be a legal adult in their country of residence.
Why is the GM1 Matrix Important?
The GM1 Matrix directly helps advance research by:
- Understanding Disease Patterns: Examining correlations between genetic variants (genotype) and clinical symptoms to better understand how GM1 gangliosidosis affects different patients
- Mapping the Diagnostic Journey: Learning about patients’ experiences getting diagnosed to help improve the process for future families
- Identifying Common Symptoms: Collecting data about symptoms across the patient population to guide research priorities
- Building Comprehensive Data: Creating an accessible GM1 Matrix dataset to share with the GM1 community and researchers
- Supporting the GM1 Community: Generating insights into the natural history of GM1 gangliosidosis by providing valuable information to researchers studying potential treatments; and building a stronger, more informed patient community
What You Need to Know About the GM1 Matrix
Oversight and Contact Information
Research Oversight: The GM1 Matrix is part of COMBINEDBrain (a non-profit research organization) which is overseen by the North Star Review Board, an independent group that reviews research studies for safety and ethics.
Questions or Concerns:
- Portal Questions: Contact the research team through the Cure GM1 Foundation, Email info@curegm1.org
- Data Withdrawal: Email studies@combinedbrain.org
- IRB Questions: Contact North Star Review Board at info@northstarreviewboard.org
Important Notes
- No Medical Advice: This study does not provide medical advice or replace consultation with your healthcare providers
- No Contact with Clinicians: Your healthcare providers will not be contacted regarding your medical records
- No Medical Record Integration: This information will never be incorporated into medical records
- International Participation: This study recruits participants from countries around the world
- Data Storage: Your data will be stored and accessed in the United States via US servers
Need Help?
Reach out to info@curegm1.org if you need assistance with:
- Technical Issues: Help with online forms or document uploads
- Questions: Understanding any part of the process
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