Changes in the Incidence of Infantile Spinal Muscular Atrophy in Shikoku, Japan between 2011 and 2020

• Main Authors: Kentaro Okamoto (Author), Hisahide Nishio (Author), Takahiro Motoki (Author), Toshihiro Jogamoto (Author), Kaori Aibara (Author), Yoichi Kondo (Author), Kentaro Kawamura (Author), Yukihiko Konishi (Author), Chiho Tokorodani (Author), Ritsuo Nishiuchi (Author), Mariko Eguchi (Author)
• Format: Book
• Published: MDPI AG, 2022-09-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder. Al-though there was no cure for SMA, newly developed therapeutic drugs (nusinersen, onasemnogene abeparvovec, and risdiplam) have been proven effective for the improvement of motor function and prevention of respiratory...