Spinocerebellar ataxia Type 1 (SCA1) is a dominantly inherited neurodegenerative disease resulting in balance, speech, thinking and coordination difficulties. SCA1 is one of the more common ataxias, with onset typically in the fourth and fifth decades of life. There is currently no treatment to slow or halt disease progression. Efforts to create and evaluate new therapies are underway.

Clinicians and scientists at Radboud University and Leiden University in the Netherlands have been running a longitudinal study looking at developing an antisense oligonucleotide therapy for SCA1, as well as examining disease progression, including changes to speech (supported by Redenlab). First year data were published earlier this year with follow up tests currently being analysed. This study will provide a clearer picture of changes that occur in the disease, setting up the use of meaningful multimodal assessments for use in future clinical trials.

Redenlab are also supporting efforts to explore speech in SCA1 across Polish, Czech, English and Turkish, with collaborators at the Institute of Psychiatry and Neurology (Warsaw), Koç University School of Medicine (Istanbul), and Neurologická klinika (Prague).

To find out more about the study, click here.