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The development of an RNA therapy for Spinocerebellar ataxia type 1 (SCA1)



WHAT WE DO?

The SCA1 research project consists of 3 parts

Observe

To accurately map the disease in patients using brain MRI, neurological testing, and assessing blood and cerebrospinal fluid. 

Test

To test the candidate therapy (the AON) in several models of SCA1.



Study

To study safety and the right dose of the candidate therapy in patients with SCA1.

This website first and foremost aims to inform patients and families that live with SCA1, but also professionals working on the disease. On this website you can find information on the disease and on the project and meet the project members. We post regular updates on the project on the news section of this website. Finally, using the contact form, you can send a message to the researchers.

This website first and foremost aims to inform patients and families that live with SCA1, but also professionals working on the disease. On this website you can find information on the disease and on the project and meet the project members. We post regular updates on the project on the news section of this website. Finally, using the contact form, you can send a message to the researchers.

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