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Clinical Trial Summary

NatHis-SMA is a prospective, longitudinal and interventional study of the natural history of patients with type 2 and 3 Spinal Muscular Atrophy (SMA). The purpose of this study is to characterize the disease course over 2 years and identify prognostic variables of the disease and biomarkers of SMA progression, as well as determine the best outcome measures for further therapeutics approaches.


Clinical Trial Description

n/a


Study Design


Related Conditions & MeSH terms


NCT number NCT02391831
Study type Interventional
Source Institut de Myologie, France
Contact
Status Completed
Phase N/A
Start date May 2015
Completion date June 2018

See also
  Status Clinical Trial Phase
Active, not recruiting NCT05089656 - Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA) Phase 3