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News and information about Spinocerebellar Ataxia research. Written by scientists in plain language that everyone can understand.

Ontario, Canada
Joined April 2018
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Could other brain cells besides neurons or nerve cells play an important role in spinocerebellar ataxia type 7 (SCA7)? A new study offers an important clue by looking beyond neurons and focusing on the brain's support cells called astrocytes. ataxia.org/scasourceposts/su…
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Before a new drug or therapy can reach patients, researchers and regulatory agencies need evidence that it is safe and effective. One of the most reliable ways to gather this evidence is through a randomized controlled trial (RCT). ataxia.org/scasourceposts/wh…
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Endpoints are clearly defined outcomes that are measured during a trial to help determine the effects of an intervention. Clinical trials usually include several types of endpoints, each serving a different purpose. ataxia.org/scasourceposts/sn…
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Levacetylleucine, sold under the brand name Aqneursa, is an FDA-approved treatment for Niemann-Pick disease type C. Levacetylleucine is also in Phase III clinical trial for Ataxia-Telangiectasia and is being studied for the treatment of other diseases. ataxia.org/scasourceposts/sn…
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