Planning future clinical trials in Machado Joseph disease: Lessons from a phase 2 trial
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- 作者：Jonas Alex Morales Sautea ; g ; Carlos R.M. Riedera ; h ; Raphael Machado Castilhosb ; i ; Thais Lampert Montea ; h ; Artur Francisco Schumacher-Schuhh ; Karina Carvalho Donisg ; Rui D'Á ; vilag ; Gabriele Nunes Souzaa ; g ; Aline Dutra Russoa ; g ; Gabriel Vasata Furtadob ; Tailise Conte Ghenob ; Diogo Onofre Gomes Souzad ; j ; Maria Luiza Saraiva-Pereirab ; d ; g ; Luis Valmor Cruz Portelad ; k ; Suzi Cameyc ; f ; Vanessa Bielefeld Leotti Tormanc ; f ; Laura Bannach Jardima ; b ; e ; g ; i ; ljardim@hcpa.edu.br" class="auth_mail" title="E-mail the corresponding author
- 关键词：Lithium ; Spinocerebellar ataxias ; Polyglutamine disorders ; Machado Joseph disease ; Treatment ; Scales
- 刊名：Journal of the Neurological Sciences
- 出版年：2015
- 出版时间：15 November 2015
- 年：2015
- 卷：358
- 期：1-2
- 页码：72-76
- 全文大小：304 K
文摘
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Primary endpoints for future trials in SCA3/MJD should focus on ataxic features.
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The inclusion of independent walking SCA3/MJD patients in future trials is advisable.
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With present outcomes, phase 3 trials in SCA3/MJD should last at least 18–24 months.