Disease modifying therapy of pediatric neuromuscular diseases / 中国小儿急救医学
Chinese Pediatric Emergency Medicine
;
(12): 405-410, 2023.
Artículo
en Chino
| WPRIM
| ID: wpr-990534
ABSTRACT
Neuromuscular diseases (NMDs) are a group of hereditary, commonly childhood-onset neurological disorders affecting various components of motor unit, which are often characterized by reduced muscle strength.With the rapid development of precision medicine, great progress has been made in the pharmacological treatment of NMDs, and pediatric NMDs has also entered the era of disease modifying therapy.Antisense oligonucleotides, gene therapy, small molecule compounds, monoclonal antibodies and other disease modifying therapy drugs targeting the cause and pathogenesis of the diseases are gradually applied in clinical practice.What′s more, a variety of potential new drugs and therapeutic biological products are in continuous development.Hopefully, new breakthroughs are expected to be achieved in the field of pediatric NMDs treatment in the near future.
Texto completo:
Disponible
Índice:
WPRIM (Pacífico Occidental)
Idioma:
Chino
Revista:
Chinese Pediatric Emergency Medicine
Año:
2023
Tipo del documento:
Artículo
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