An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients with Hemophilia B (CSL222_4001)

ENCePP Code of conduct

No

Data sources

• Data source(s), other:
  ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders (Hemophilia Cohort, Gene Therapy Outcomes Arm and Natural History Arm), United States
• 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