A registry-based cohort study of Spinal Muscular Atrophy (SMA) disease to describe the natural history of SMA, the evolution of SMA care management and disease progression considering new disease modifying therapies (DMTs).

ENCePP Code of conduct

No

Data sources

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

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