To better understand the factors that determine disease progression among cerebellar ataxia with the goal of improving the understanding of the disease process, current treatments, and development of disease-modifying therapies
Learn more about how you can get involved in the CRC-SCA Natural History Study.
General information for researchers working with Ataxia.
Introductory information for industry partners that are exploring Ataxia therapies.
For PI’s and Coordinators of CRC-SCA Sites.
A natural history study collects data that shows how a specific disease progresses in individuals over time. Natural history studies are essential for developing clinical trial designs that will facilitate drug development. The natural history of the SCAs and factors that may modify them are not completely understood. There is a need to define natural history in geographically distinct areas and develop additional methods to document progression that may be more sensitive and reliable. There is also limited knowledge of factors that may modify symptoms of SCA.
The CRC-SCA natural history study and biomarker development is growing and adapting during this exciting new era in ataxia research as pharmaceuticals are advancing to therapy development for the ataxias. CRC-SCA plans to add specific clinical end-point assessments and biospecimen collection. This will increase the strength of this study allowing the consortium to partner with stakeholders in a meaningful way that will include patients as research collaborators.
The Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA) continues to recruit research participants who have a confirmed diagnosis of SCA 1, 2, 3, 6, 7, 8 or 10. This is an opportunity for anyone in the United States with those forms of SCA at any stage of the disease to participate. Contact the research coordinator at a site near you to learn more about how you might be able to help in Ataxia research efforts to discover a treatment.
Liana Rosenthal, MD, PhD
Johns Hopkins University,
Vikram Shakkottai, MD, PhD
UT Southwestern Medical Center,
Sheng-Han Kuo, MD
New York City, NY
Montréal, Québec, Canada
NAF extends its appreciation to the Gordon and Marilyn Macklin Foundation for awarding NAF a matching grant that provides support for the sites across the country who are recruiting participants in the Natural History Study. This clinical research collects the essential data needed to provide our industry partners information about disease progression which guides them as they work to develop treatments and design clinical trials. This effort is paramount to make the necessary advancements in pursuit of a treatment for Ataxia and, ultimately, a cure.