Disease modifying therapy of pediatric neuromuscular diseases
10.3760/cma.j.issn.1673-4912.2023.06.002
- VernacularTitle:儿童神经肌肉病的疾病修正治疗
- Author:
Shanshan MAO
1
;
Siyi HUANG
Author Information
1. 浙江大学医学院附属儿童医院神经内科 国家儿童健康与疾病临床医学研究中心，杭州 310052
- Keywords:
Neuromuscular diseases;
Disease modifying therapy;
Children
- From:
Chinese Pediatric Emergency Medicine
2023;30(6):405-410
- CountryChina
- Language:Chinese
-
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.
