READISCA Data

Description

READISCA is an NIH-funded international clinical trial readiness study for spinocerebellar ataxia type 1 (SCA1) and type 3 (SCA3). These SCAs belong to a group of ~50 autosomal dominant diseases that cause degeneration of the cerebellum and variable other parts of the nervous system. Patients with these SCAs suffer from progressive loss of balance, hand incoordination, slurred speech and additional features leading to eventual total disability and premature death. There are no efficacious treatments other than supportive therapies. Advances in research into disease mechanisms in SCAs have resulted in the development of rational experimental treatments. Early therapeutic interventions have shown better efficacy than treatments in the later stages in genetic animal models of these diseases and clinical trials of related disorders. Clinical trials of these therapeutic interventions are starting now. In READISCA we have obtained critical information to optimally design such clinical trials and correctly interpret the results in pre-ataxic and early ataxic carriers of SCA1 and SCA3 mutations. This longitudinal multi-center study began in 2018 and data acquisition was completed in 2023. The study was co-funded by NIH (U01 NS104326; baseline, 1-year and 2-year follow-up visits) and Biogen, Inc. (6 month and 3-year follow up visits). Further information about the study can be found at the READISCA site.

Researchers interested in neurodegenerative diseases can now access de-identified clinical and MRI data collected under the READISCA project upon agreeing to a set of Terms and Conditions. Recipient investigators are asked to agree to restrictions against attempting to identify study participants, restrictions on redistribution of the data to third parties, and to properly acknowledge the READISCA team and funding sources.