Pharmacotherapy for the management of the symptoms of Machado-Joseph Disease.

INTRODUCTION: Machado-Joseph disease or spinocerebellar ataxia type 3 (SCA3/MJD) is the leading cause of autosomal dominant ataxia worldwide. This is a slowly progressive, but very disabling disorder. Ataxia is the main clinical feature, but additional motor and non-motor manifestations may be found. Many of these manifestations are amenable to pharmacological treatments, which may impact the quality of life of affected subjects. AREAS COVERED: Authors review available literature on both disease-modifying and symptomatic pharmacological therapies for SCA3/MJD. Discussion is stratified into motor (ataxic and non-ataxic syndromes) and non-motor manifestations. Ongoing clinical trials and future perspectives are also discussed in the manuscript. EXPERT OPINION: Symptomatic treatment is the mainstay of clinical care and should be tailored for each patient with SCA3/MJD. Management of ataxia is still a challenging task, but relief (at least partial) of dystonia, pain/cramps, fatigue, and sleep disorders is an achievable goal for many patients. Even though there are no disease-modifying treatments so far, recent advances in understanding the biology of disease and international collaborations of clinical researchers are now paving the way for a new era where more clinical trials will be available for this devastating disorder.

Objectively measured daily-life physical activity of moderate-to-severe Brazilian asthmatic women in comparison to healthy controls: A cross-sectional study.

Considering the complex relationship between asthma symptoms and exercise, asthmatics are usually believed to be less active in daily life than healthy subjects. However, few studies have objectively assessed daily-life physical activity (DLPA) of asthmatic adults. OBJECTIVE: To objectively assess DLPA of a sample of Brazilian asthmatic women in comparison to healthy controls, and to investigate the associations between DLPA and asthma control, health-related quality of life, anxiety and depression levels, and the Six-minute walk test (6MWT) in this population. METHODS: Sixty-six women were included, 36 in the asthma group (AG) and 30 in the control group (CG). The AG was composed by clinically stable moderate-to-severe asthmatics. The CG was composed by apparently healthy volunteers. All subjects underwent DLPA assessment (considered as the average of steps taken during six consecutive days measured by a pedometer) and performed the 6MWT. Additionally, participants in the AG were assessed using the Asthma Control Questionnaire, the Asthma Quality of Life Questionnaire (AQLQ), and the Hospital Anxiety and Depression Scale. RESULTS: There was no difference between the AG and the CG regarding DLPA (7490.3 ± 3330.2 vs 6876.4 ± 3242.1 steps respectively, p = 0.45), even after adjustment for covariates. DLPA was significantly correlated to the activity limitation domain of the AQLQ among asthmatics (r = 0.43, p < 0.01). CONCLUSION: Despite the association between self-perceived activity limitation and DLPA among asthmatics, there were no differences regarding DLPA between a sample of moderate-to-severe Brazilian asthmatic women and apparently healthy controls.