Pharmacological Therapies of Spinal Muscular Atrophy: A Narrative Review of Preclinical, Clinical–Experimental, and Real-World Evidence

Spinal muscular atrophy (SMA) is a rare neuromuscular disease, with an estimated incidence of about 1 in 10,000 live births. To date, three orphan drugs have been approved for the treatment of SMA: nusinersen, onasemnogene abeparvovec, and risdiplam. The aim of this narrative review was to provide a...

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Bibliographic Details

Published in:	Brain Sciences
Main Authors:	Salvatore Crisafulli, Brigida Boccanegra, Giacomo Vitturi, Gianluca Trifirò, Annamaria De Luca
Format:	Article
Language:	English
Published:	MDPI AG 2023-10-01
Subjects:	spinal muscular atrophy nusinersen onasemnogene abeparvovec risdiplam pre-marketing evidence post-marketing evidence
Online Access:	https://www.mdpi.com/2076-3425/13/10/1446