Transforming Spinal Muscular Atrophy: From Pivotal Trials to Real-World Evidence and Future Therapeutic Frontiers in Types 1 and 2

Spinal muscular atrophy (SMA) is a rare, autosomal recessive neuromuscular disorder and a leading genetic cause of infant mortality. The past decade has witnessed a paradigm shift in SMA management with the advent of disease-modifying drugs (DMDs). This narrative review aims to (i) summarize pivotal...

詳細記述

書誌詳細

出版年:	Biomedicines
主要な著者:	Andrej Belančić, Patrick Castillo Eustaquio, Elvira Meni Maria Gkrinia, Valentino Rački, Kristina Pilipović, Dinko Vitezić
フォーマット:	論文
言語:	英語
出版事項:	MDPI AG 2025-08-01
主題:	spinal muscular atrophy SMA disease-modifying drugs nusinersen risdiplam onasemnogene abeparvovec
オンライン･アクセス:	https://www.mdpi.com/2227-9059/13/8/1939