Machado Joseph disease: clinical and genetic aspects, and current treatment

JAM Saute, LB Jardim - Expert Opinion on Orphan Drugs, 2015 - Taylor & Francis
Introduction: Spinocerebellar ataxia (SCA) type 3/Machado–Joseph disease (SCA3/MJD) is
the most common SCA worldwide. SCA3/MJD is an adult onset cerebellar ataxia associated …

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

JAM Saute, CRM Rieder, RM Castilhos… - Journal of the …, 2015 - Elsevier
Background In a recent phase 2 clinical trial in spinocerebellar ataxia type 3/Machado
Joseph disease (SCA3/MJD), a neurogenetic disorder without specific therapy, benefits of …

Modifications of resting state networks in spinocerebellar ataxia type 2

S Cocozza, F Saccà, A Cervo, A Marsili… - Movement …, 2015 - Wiley Online Library
Purpose We aimed to investigate the integrity of the Resting State Networks in
spinocerebellar ataxia type 2 (SCA2) and the correlations between the modification of these …

Towards a therapeutic intervention in polyglutamine ataxias: from models to clinical trials

N Kazachkova, M Lima - Front Clin Res-CNS Neurolog Disord, 2015 - books.google.com
The autosomal dominant (AD) forms of hereditary ataxias compose a heterogeneous group
of diseases, in which cerebellar degeneration and dysfunction is consistently present …

[引用][C] Modifications of Resting State Networks in Spinocerebellar Ataxia Type 2

SM delle Acque Giorgio, MD Giuseppe De Michele… - Age (mean 6 SD)