Evolving therapies in neuronopathic LSDs: opportunities and challenges.
Metab Brain Dis
; 37(7): 2245-2256, 2022 10.
Article
em En
| MEDLINE
| ID: mdl-35442005
ABSTRACT
Lysosomal storage disorders (LSD) are multisystemic progressive disorders caused by genetic mutations involving lysosomal function. While LSDs are individually considered rare diseases, the overall true prevalence of these disorders is likely higher than our current estimates. More than two third of the LSDs have associated neurodegeneration and the neurological phenotype often defines the course of the disease and treatment outcomes. Addressing the neurological involvement in LSDs has posed a significant challenge in the rapidly evolving field of therapies for these diseases. In this review, we summarize current approaches and clinical trials available for patients with neuronopathic lysosomal storage disorders, exploring the opportunities and challenges that have emerged with each of these.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Doenças por Armazenamento dos Lisossomos
Tipo de estudo:
Risk_factors_studies
Limite:
Humans
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article