This paper is in the following e-collection/theme issue:

Non-Randomized Study Protocols and Methods (Non-eHealth) (683) Demographics of Users, Social & Digital Divide (913)

Published on in Vol 11, No 12 (2022): December

Preprints (earlier versions) of this paper are available at https://preprints.jmir.org/preprint/38878, first published .
The Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA): Protocol for a Longitudinal Observational Study

The Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA): Protocol for a Longitudinal Observational Study

The Japan Registry for Adult Subjects of Spinal Muscular Atrophy (jREACT-SMA): Protocol for a Longitudinal Observational Study

Authors of this article:

Kentaro Sahashi1 Author Orcid Image ;   Atsushi Hashizume2 Author Orcid Image ;   Yachiyo Kuwatsuka3 Author Orcid Image ;   Madoka Chinen4 Author Orcid Image ;   Ai Saotome-Nakamura4 Author Orcid Image ;   Masahiko Ando3 Author Orcid Image ;   Masahisa Katsuno1, 2 Author Orcid Image
Article Authors Cited by (6) Tweetations (2) Metrics

Journals

  1. Tachibana Y, Sato R, Makioka H, Hoshino M, Jin M. Safety and effectiveness of nusinersen, a treatment for spinal muscular atrophy, in 524 patients: results from an interim analysis of post-marketing surveillance in Japan. International Journal of Neuroscience 2024;134(11):1185 View
  2. Yao X, Peng J, Luo R, Wang X, Lu X, Wu L, Jin R, Zhong J, Liang J, Hong S, Yang L, Zhang X, Mao S, Hu J, Tao Z, Sun D, Wang H, Zhang L, Xia Y, Chen K, Wang Y. Nusinersen effectiveness and safety in pediatric patients with 5q-spinal muscular atrophy: a multi-center disease registry in China. Journal of Neurology 2024;271(8):5378 View
  3. Mak C, Lee J, Wong J, Law C, Chan C, Yeung C, Pang S, Hau E, Chan S. Views of the Public and Healthcare Professionals on Newborn Screening for Spinal Muscular Atrophy and the Potential for Detecting Adult-Onset Types in Patients-in-Waiting in Hong Kong. Cureus 2025 View
  4. Balaji L, Farrar M, Yiu E, Kariyawasam D. A state-of-the-art review of registries in spinal muscular atrophy: A valuable resource for clinical research. Journal of Neuromuscular Diseases 2025;12(3):312 View
  5. Puig-Ram C, Segovia S, Garcia-Uzquiano R, Ñungo Garzón N, Aragon-Gawinska K, García Romero M, Expósito-Escudero J, Carrera-García L, López-Lobato M, Paradas C, González Mera L, Álvarez Molinero M, Gómez Andrés D, Toro E, Fernández Ramos J, Grimalt M, Toledo Bravo de Laguna L, González Barrios D, Tizzano E, Cattinari M, Medina J, Calvo Medina R, Munell F, Sotoca J, Martínez-Salcedo E, Moreno Escribano A, Povedano Panadés M, Fernández-García M, Pitarch-Castellano I, Vázquez-Costa J, Natera-de Benito D, Nascimento A. Real-world data on spinal muscular atrophy in Spain: Insights from over 500 individuals in the CuidAME project. Journal of Neuromuscular Diseases 2025;12(6):837 View
  6. Sahashi K, Katsuno M. 4) Antisense Therapeutics for Spinal Muscular Atrophy. Nihon Naika Gakkai Zasshi 2024;113(9):1581 View