Impact of Nusinersen on the Health-Related Quality of Life and Caregiver Burden in Patients with Spinal Muscular Atrophy with Symptom Onset before the Age of 6 Months

Purpose This study investigated the impact of nusinersen on health-related quality of life (HRQoL), functional performance, and caregiver burden in patients with infantile-onset spinal muscular atrophy (SMA), addressing a growing interest in disease-modifying treatments. Methods A 14-month observati...

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Autores principales:	Yun Jeong Lee, Hyunwoo Bae, Young Kyu Shim, Jae So Cho, Jong Hee Chae, Soonhak Kwon
Formato:	Artigo
Lenguaje:	Inglês
Publicado:	Korean Child Neurology Society 2025-04-01
Colección:	Annals of Child Neurology
Materias:	spinal muscular atrophies of childhood nusinersen caregiver burden quality of life
Acceso en línea:	http://annchildneurol.org/upload/pdf/acn-2024-00668.pdf
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