An overview of the Cure SMA membership database: Highlights of key demographic and clinical characteristics of SMA members

Lisa Belter, Suzanne F. Cook, Thomas O. Crawford, Jill Jarecki, Cynthia C. Jones, John T. Kissel, Mary Schroth, Kenneth Hobby

Research output: Contribution to journalArticlepeer-review

Abstract

Background: The Cure SMA database is one of the largest patient reported databases for people affected with SMA. Objective: The purpose of this study was to examine a subset of affected SMA persons with types I, II, and III from a patient reported database. Methods: Individuals with SMA were selected from the database using a date of first contact to Cure SMA between 2010 and 2016. Data analyzed included age at diagnosis, number of weeks from SMA diagnosis to contacting Cure SMA, and geographic distribution of individuals. Results: A total of 1,966 individuals with SMA were included in the analysis. Of these individuals, 51.9% had type I, 32.3% had type II, and 15.8% had type III. The average age of diagnosis for type I patients was 5.2 months, 22.1 months for type II, and 97.8 months for type III. From published incidence, about 59.0% of affected individuals in the US are registered in the Cure SMA database. Conclusions: The Cure SMA database is a unique and robust source of patient reported data that informs on the burden of illness and supports the development of new therapeutic modalities.

Original languageEnglish (US)
Pages (from-to)167-176
Number of pages10
JournalJournal of neuromuscular diseases
Volume5
Issue number2
DOIs
StatePublished - 2018

Keywords

  • Spinal muscular atrophy
  • birth prevalence
  • nusinersen
  • patient registries
  • patient reported data
  • survival

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology

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