Other rare diseases and orphan drugs

  • Literature review, consensus meeting and mapping of the treatment pathway in adrenoleukodystrophy
  • Presentation on the challenges and solutions of selecting and developing endpoints for use in paediatric populations and rare diseases
  • Measurement strategy to demonstrate efficacy of medical devices in patients with pigmented villonodular synovitis (PVNS) or tenosynovial giant cell tumour (dTGCT)

Related categories

Experience,  Orphan drugs,  Special interest