Later efficacy of nusinersen treatment in adult patients with spinal muscular atrophy: A retrospective case study with a median 4-year follow-up

BACKGROUNDSpinal muscular atrophy (SMA) is a hereditary neuromuscular disorder characterized by skeletal muscle atrophy and weakness. New treatments for SMA have been developed namely, the drugs nusinersen, onasemnogene abeparvovec, and risdiplam. However, there are limited reports on their effects...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Brain & development 2024, Vol.46 (1), p.62-67
Hauptverfasser:	Funato, Michinori, Kino, Atsunari, Iwata, Reina, Yumioka, Misaki, Yamashita, Kohei, Urui, Chika, Uno, Ryoya, Kondo, Emi, Morioka, Etsuko, Ogawa, Yoko, Kawamura, Akihisa, Kusukawa, Toshifumi, Minatsu, Hiroshi
Format:	Report
Sprache:	eng
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!