A Prospective, Observational, Post-Authorisation Efficacy Study to Assess Long-term Effectiveness of Risdiplam in Patients with Genetically Confirmed 5q SMA

ENCePP Code of conduct

No

Data sources

• Data source(s):
  Translational Research in Europe - Assessment and Treatment of Neuromuscular Diseases

  Longitudinal Data Collection from Patients with Spinal Muscular Atrophy (SMArtCARE)
• Data source(s), other:
  SMA Research and Clinical Hub (REACH UK), French Register of Patients with Spinal Muscular Atrophy (R-SMA France), Neuromuscular Diseases in Sweden – Neuromuskulära Sjukdomar i Sverige (NMiS), The Australian Neuromuscular Disease Registry, The Canadian Neuromuscular Disease Registry, CureSMA: 3 databases: CureSMA Membership Database, Pediatric Neuromuscular Clinical Research (PNCR), and the Clinical Care Network
• Data sources (types):
  Disease registry

  Other
• Data sources (types), other:
  Prospective patient-based data collection

Use of a Common Data Model (CDM)

• CDM mapping:
  No

Data quality specifications

• Check conformance:
  Unknown
• Check completeness:
  Unknown
• Check stability:
  Unknown
• Check logical consistency:
  Unknown

Data characterisation

• Data characterisation conducted:
  No