Clinical Research Consortium for the
Study of Cerebellar Ataxia
Natural History Study – Why is it so Important?
One of the challenges in developing a treatment for a rare disease, such as Ataxia, is the lack of natural history studies. 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.
With the recent funding by the National Ataxia Foundation of the Clinical Research Consortium for the Study of Cerebellar Ataxia (CRC-SCA), a natural history study will begin for SCAs 1, 2, 3, 6 and 7 (and SCA 8 and 10 at some sites). The institutions and principle investigators that are in the CRC-SCA are listed below:
- Columbia University – Dr. Sheng Han Kuo
- Emory University – Dr. George (Chip) Wilmot
- Houston Methodist Research Institute – Dr. Tetsuo Ashizawa
- Johns Hopkins University – Drs. Liana Rosenthal and Chiadikaobi Onyike
- Massachusetts General – Dr. Jeremy Schmahmann
- Northwestern University – Dr. Puneet Opal
Contact: Karen Williams 312-503-2068
- University of California – Dr. Susan Perlman
- University of Chicago – Dr. Christopher Gomez
- University of Florida – Dr. SH Subramony
- University of Michigan – Drs. Henry Paulson and Vikram Shakkottai
- University of Minnesota – Dr. Khalaf Bushara
- University of Utah – Dr. Stefan Pulst
The success of the natural history study is dependent on participation by individuals who have those forms of SCA. If you have SCA 1, 2, 3, 6 or 7 (and SCA 8 and 10 at some sites) and are a patient at one of those sites or can travel to a site, please contact the clinic office for more information. You may also contact Sue Hagen at NAF at 763-231-2742 or email@example.com for more information.
Ataxia Patient Registry – Why is it so important?
The purpose of a patient registry is to advance the development of treatments for a disease by connecting researchers with those who are affected with that disease. The National Ataxia Foundation partners with the Coordination of Rare Diseases at Sanford (CoRDS) to enroll those who are affected with Ataxia or are at-risk for Ataxia in the Ataxia Patient Registry. Currently over 1,000 people have enrolled in the Ataxia registry. Three researchers have requested access to de-identified data so that CoRDS can inform enrollees of their research studies. Anyone affected or at-risk for Ataxia are encouraged to enroll in CoRDS. Use this link to enroll or if you prefer to enroll by mail, please contact CoRDS personnel directly at 1-877-658-9192.