Pharmacotherapy for the management of the symptoms of Machado-Joseph Disease
| Autor: | Jessica Blanc Leite, Oliveira, Alberto R M, Martinez, Marcondes Cavalcante, França |
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| Rok vydání: | 2022 |
| Předmět: | |
| Zdroj: | Expert Opinion on Pharmacotherapy. 23:1687-1694 |
| ISSN: | 1744-7666 1465-6566 |
| DOI: | 10.1080/14656566.2022.2135432 |
| Popis: | 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.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.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. |
| Databáze: | OpenAIRE |
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