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...

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Detalhes bibliográficos

Principais autores:	Andrej Belančić, Patrick Castillo Eustaquio, Elvira Meni Maria Gkrinia, Valentino Rački, Kristina Pilipović, Dinko Vitezić
Formato:	Artigo
Idioma:	Inglês
Publicado em:	MDPI AG 2025-08-01
Colecção:	Biomedicines
Assuntos:	spinal muscular atrophy SMA disease-modifying drugs nusinersen risdiplam onasemnogene abeparvovec
Acesso em linha:	https://www.mdpi.com/2227-9059/13/8/1939
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