Long‐Term Effects of Nusinersen Dosing Frequency on Adult Patients With Spinal Muscular Atrophy: Efficacy of a 6‐Month Dosing Interval

ABSTRACT Objective Spinal muscular atrophy (SMA) is a genetic disease caused by the degeneration of spinal motor neurons due to a deficiency in survival motor neuron protein (SMN) protein, leading to progressive muscle atrophy and weakness. nusinersen, an antisense oligonucleotide that increases SMN...

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Hlavní autoři:	Keita Takahashi, Hitaru Kishida, Misako Kunii, Yosuke Miyaji, Yuichi Higashiyama, Hiroshi Doi, Naohisa Ueda, Hideyuki Takeuchi, Fumiaki Tanaka
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Wiley 2025-05-01
Edice:	Brain and Behavior
Témata:	adult patient long‐term efficacy nusinersen spinal muscular atrophy (SMA) survival motor neuron protein (SMN)
On-line přístup:	https://doi.org/10.1002/brb3.70528
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