Planning future clinical trials in Machado Joseph disease: Lessons from a phase 2 trial

Journal of the Neurological Sciences

Volumn 358, Issue 1-2, 2015, Pages 72-76

  Saute, Jonas Alex Morales ^a,b   Rieder, Carlos R M ^a,b   Castilhos, Raphael Machado ^a,c   Monte, Thais Lampert ^a,b   Schumacher Schuh, Artur Francisco ^b   Donis, Karina Carvalho ^b   D'Ávila, Rui ^b   Souza, Gabriele Nunes ^a,b   Russo, Aline Dutra ^a,b   Furtado, Gabriel Vasata ^a   Gheno, Tailise Conte ^a   Souza, Diogo Onofre Gomes ^a,d   Saraiva Pereira, Maria Luiza ^a,b   Portela, Luis Valmor Cruz ^a,d   Camey, Suzi ^a   Torman, Vanessa Bielefeld Leotti ^a   Jardim, Laura Bannach ^a,b,c  

^a FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^b HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^c INAGEMP Instituto Nacional de Genetica Medica Populacional   (Brazil)

^d Instituto Nacional de Ciência e Tecnologia em Excitotoxicidade e Neuroprotec õ   (Brazil)

Author keywords

Lithium; Machado Joseph disease; Polyglutamine disorders; Scales; Spinocerebellar ataxias; Treatment

Indexed keywords

LITHIUM CARBONATE; PLACEBO; ENZYME INHIBITOR;

ARTICLE; CLINICAL ARTICLE; COHORT ANALYSIS; COMPOSITE CEREBELLAR FUNCTIONAL SCORE; CONTROLLED STUDY; DOUBLE BLIND PROCEDURE; HUMAN; MACHADO JOSEPH DISEASE; NEUROLOGICAL EXAMINATION SCORE FOR THE ASSESSMENT OF SPINOCEREBELLAR ATAXIA; OUTCOME ASSESSMENT; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SCALE FOR THE ASSESSMENT AND RATING OF ATAXIA; SCORING SYSTEM; SPINOCEREBELLAR ATAXIA FUNCTIONAL INDEX; SPINOCEREBELLAR DEGENERATION; TREATMENT DURATION; TREATMENT PLANNING; TREATMENT RESPONSE; WALKING; CLINICAL TRIAL (TOPIC); MACHADO-JOSEPH DISEASE; METHODOLOGY; PHASE 2 CLINICAL TRIAL (TOPIC); STANDARDS;

CLINICAL TRIALS AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; DOUBLE-BLIND METHOD; ENZYME INHIBITORS; HUMANS; LITHIUM CARBONATE; MACHADO-JOSEPH DISEASE; OUTCOME ASSESSMENT (HEALTH CARE); RESEARCH DESIGN;

EID: 84956754972     PISSN: 0022510X     EISSN: 18785883     Source Type: Journal    
DOI: 10.1016/j.jns.2015.08.019     Document Type: Article

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