Unraveling the Heterogeneity of ALS-A Call to Redefine Patient Stratification for Better Outcomes in Clinical Trials.
Cells
; 13(5)2024 Mar 05.
Article
em En
| MEDLINE
| ID: mdl-38474416
ABSTRACT
Despite tremendous efforts in basic research and a growing number of clinical trials aiming to find effective treatments, amyotrophic lateral sclerosis (ALS) remains an incurable disease. One possible reason for the lack of effective causative treatment options is that ALS may not be a single disease entity but rather may represent a clinical syndrome, with diverse genetic and molecular causes, histopathological alterations, and subsequent clinical presentations contributing to its complexity and variability among individuals. Defining a way to subcluster ALS patients is becoming a central endeavor in the field. Identifying specific clusters and applying them in clinical trials could enable the development of more effective treatments. This review aims to summarize the available data on heterogeneity in ALS with regard to various aspects, e.g., clinical, genetic, and molecular.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Esclerose Lateral Amiotrófica
Limite:
Humans
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article