There are many ways to contribute to research through sharing your experiences. Understanding the progression of these diseases is critical to the development of a clinical trial. You can play a role by participating in natural history studies, registries and other data collection studies. If you are considering participating in a research study, you are welcome to call NTSAD to discuss the benefits and risks. Below are current ways to contribute.
AllStripes for GM1 and GM2 Gangliosidosis
You can accelerate drug development in GM2 (Tay-Sachs and Sandhoff). Important research records are trapped in hospitals across the country. As a result, drug researchers can’t access the information they need to advance treatments. But as a patient, you can. Unlock your records and contribute them to research, privately and securely.
A natural history study for Canavan disease sponsored by Aspa Therapeutics. To learn how you can enroll for this study, visit the clinicaltrials.gov posting here.
This study will collect patient-specific home-based video data and qualitative interviews with caregivers of children with GM1 Gangliosidosis. To learn more about taking part, visit the posting on clinicaltrials.gov here.
This natural history study for GM1 and GM2 (Tay-Sachs and Sandhoff) will provide a better understanding of the natural course of these diseases. Visit here for more information on how to enroll at clinicaltrials.gov.
Patient Insights Network (PIN)
The following PINs have been set up in collaboration with Canavan Research Illinois, the Canavan Foundation and the Cure GM1 Foundation respectively. A GM2 (Tay-Sachs, Sandhoff) will be established soon as a collaboration with the Cure Tay-Sachs Foundation.
Canavan Patient Insights Network (PIN) - Join here.
GM1 Patient Insights Network (PIN) - Join here.
GM2 Patient Insights Network (PIN) - Join here.
PRONTO - Azafaros
A natural history study of GM1 and GM2 (Sandhoff and Tay-Sachs)
Clinical Trial #NCT05109793
Data about the progression of neurological manifestations in the late-infantile or juvenile onset forms of GM1 and GM2 is limited to single patient case reports and small case studies. Taking part in a Natural History study will help collect data in a systematic way, with measurements harmonies between the participating centers. These studies will help in the design of future studies with new future treatments in a more rigorous scientific way.
View video about the study here in either Brazilian Portgueses, French, German, Italian, English (UK), English (United States).