Article
Survey of patients with spinal muscular atrophy on the island of Shikoku, Japan.
Brain & development - 1 Sept 2020
Okamoto Kentaro, Motoki Takahiro, Saito Isao, Urate Risako, Aibara Kaori, Jogamoto Toshihiro, Fukuda Mitsumasa, Wakamoto Hiroyuki, Maniwa Satoshi, Kondo Yoichi, Toda Yoshihiro, Goji Aya, Mori Tatsuo, Soga Tomohiro, Konishi Yukihiko, Nagai Shigehiro, Takami Yoko, Tokorodani Chiho, Nishiuchi Ritsuo, Usui Daisuke, Ando Rina, Tada Satoshi, Yamanishi Yuki, Nagai Masahiro, Arakawa Reiko, Saito Kayoko, Nishio Hisahide, Ishii Eiichi, Eguchi Mariko
Abstract excerpt
BACKGROUND: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder associated with spinal motor neuron loss and characterized by generalized muscle weakness. Only a few reports exist on SMA epidemiology in Japan. Additionally, nusinersen recently became available as a treatment for this condition. We estimated the prevalence of each type of SMA on Shikoku, Japan's fourth-largest major island....
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