Journal of neuromuscular diseases
Characteristics of early-onset, rapidly progressive scoliosis in spinal muscular atrophy type I treated with disease-modifying therapy -a multicenter retrospective study conducted in Japan.
Tomokazu Kimizu, Reiko Arakawa, Mikiko Hasegawa, Tomoko Mizuno, Ryosuke Bou, Emiko Kobayashi, Toshio Saito, Kazuhiro Muramatsu, Yoshi-Ichiro Kamijo, Tamaki Kato, Kenji Inoue, Mitsuo Motobayashi, Yuichi Abe, Keisuke Oki, Saki Yokawa
Published: 202610.1177/22143602251414327
Abstract
In the era of disease-modifying therapy (DMT), almost all patients with spinal muscular atrophy (SMA) type I treated after onset, but before 6 months of age, develop early-onset, rapidly progressive scoliosis by 2 years of age, despite improvements i…
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