Unmet needs and evolving treatment for limb girdle muscular dystrophies.
Neurodegener Dis Manag
; 11(5): 411-429, 2021 10.
Article
en En
| MEDLINE
| ID: mdl-34472379
Lay abstract Limb-girdle muscular dystrophy is a term that is applied to a group of relatively rare forms of muscular dystrophy. The term 'LGMD' was introduced in the 1950's, but there were no strict rules for defining the condition. This changed as a result of the 229th European Neuromuscular Center International Workshop in 2017 providing a clear definition and classification discussed in this article. Limb-girdle muscular dystrophy is now recognized as a genetic muscle disease with an elevated serum creatine kinase and dystrophic changes on muscle histology. Most treatments up to now rely on supportive measures for heart and lungs and assisting the physical limitations. Medications have not proven to be beneficial to stop progression of disease. This article focuses on new innovations of treatment that target the effected gene and the use special methods to replace the abnormal gene.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Distrofia Muscular de Cinturas
/
Sarcoglicanopatías
Límite:
Humans
Idioma:
En
Revista:
Neurodegener Dis Manag
Año:
2021
Tipo del documento:
Article
País de afiliación:
Estados Unidos