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A new target to treat spinocerebellar ataxia ?

Spinocerebellar ataxias are genetic neurodegenerative diseases of the cerebellum and brainstem causing numerous motor disorders, for which no curative or preventive therapies are available. SCA3 or Machado-Joseph disease (MJD) is the most prevalent ataxia worldwide. In their work published on June 14 in Acta Neuropathologica, Nathalie Cartier-Lacave, researcher from Inserm at the Brain and Spine Institute and NeurATRIS member, and her team have discovered the crucial role of an enzyme allowing to alleviate symptoms of the disease in mice.

Read the complete press release on the ICM website (in French): https://icm-institute.org/fr/actualite/nouvelle-cible-therapeutique-traiter-ataxies-spinocerebelleuses/

Scientific Publication:

RESTORING BRAIN CHOLESTEROL TURNOVER IMPROVES AUTOPHAGY AND HAS THERAPEUTIC POTENTIAL IN MOUSE MODELS OF SPINOCEREBELLAR ATAXIA.

Nóbrega C, Mendonça L, Marcelo A, Lamazière A, Tomé S, Despres G, Matos CA, Mechmet F, Langui D, den Dunnen W, de Almeida LP, Cartier N, Alves S.

Acta Neuropathol. 2019 Jun 14.