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A randomized, phase 2 clinical trial of lithium carbonate in Machado-Joseph disease.
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Mov Disord 2014 Jan;
Authors: Jonas Alex Morales Saute, Raphael Machado de Castilhos, Thais Lampert Monte, Artur Francisco Schumacher-Schuh, Karina Carvalho Donis, Rui D'Ńvila, Gabriele Nunes Souza, Aline Dutra Russo, Gabriel Vasata Furtado, Tailise Conte Gheno, Diogo Onofre Gomes de Souza, Luis Valmor Cruz Portela, Maria-Luiza Saraiva-Pereira, Suzi Alvez Camey, Vanessa Bielefeld Leotti Torman, Carlos Roberto de Mello Rieder, Laura Bannach Jardim
Because lithium exerts neuroprotective effects in preclinical models of polyglutamine disorders, our objective was to assess the safety and efficacy of lithium carbonate (0.5-0.8 milliequivalents per liter) in patients with Machado-Joseph disease (spinocerebellar ataxia type 3 [MJD/SCA3]).
PMID: 24399647 [PubMed - as supplied by publisher]