Participate in Research and Data Sharing

Cure GM1 presently has three initiatives to support natural history and sample collection for GM1.

REAL-WORLD EVIDENCE NATURAL HISTORY, DATA SHARING aND BIOBANK

In order to support drug development and research, Cure GM1 has multiple initiatives that support research and drug development. Families can participate in these efforts to ensure that GM1 is research-ready!

From the research perspective, these are very separate endeavors, both in design and deliverables, but their shared purpose is to gather and share data from many patients to inform research and advance the knowledge of GM1, so that ultimately those impacted by GM1 can receive better care.

These are critically important building blocks in the drug development process.

XCURES REAL-WORLD EVIDENCE NATURAL HISTORY

xCures is a company that consolidates medical records from anywhere, normalizing and structuring them into a comprehensive, searchable dataset of all of a patient’s clinical data. This collaboration is lead by Cure GM1 and is a study which is approved by regulatory authorities. A pilot study is now being completed and we aim to expand this effort in the future. While the company’s focus has been in oncology, the technology and platform can be applied to rare diseases such as GM1.

This study is IRB-approved and listed on clinicaltrials.gov.

The advantage of this approach is that the electronic health records (EHR) can be collected with appropriate consent without requiring visits in-person and can also be used to collect historical and current data.

GM1 NATURAL HISTORY DATA SHARING INITIATIVE

The aim of this initiative is to build a forum for collaboration and aggregation of natural history data that will increase knowledge of GM1 and establish meaningful clinical endpoints and a comparator control dataset suitable for clinical treatment trials and regulatory filings. The Cure GM1 Foundation founded a global collaborative group, The GM1 Natural History Data Sharing Collaborative, a network of disease experts, organizations, and individuals conducting natural history studies in GM1.

To learn more, you can see our recent poster on this critically imporant initiative. This collaboration involves many entities such as the Critical Path Institute and universities with natural history data. This effort aims to reduce the need for new natural history studies and to reduce waste of resources and time.

COMBINED BRAIN bIOREPOSITORY AND COLLABORATIVE RESEARCH APPROACH

CombinedBrain is a Consortium for Outcome Measures and Biomarkers for Neurodevelopmental Disorders. This organization is a nonprofit devoted to speeding the path to clinical treatments for people with rare genetic neurological disorders by pooling efforts, studies, and data. CombinedBrain has a biorepository and services which enable patient advocacy groups to collect and direct research using biospecimens. Biospecimens can even be contributed from home. Researchers may also request access to the samples.

CONTRIBUTE TO RESEARCH AND PARTICIPATE NOW

If you are the caregiver for someone with GM1, please consider participating these initiatives. You can always email info@curegm1.org to request a call to discuss your participation. The more data that becomes available, the faster treatment can be developed. Affected families can help our cause enormously.

Please visit our Take Action Page to learn about what else you can do to help Cure GM1.