Mucopolysaccharidosis

  • Understanding the experience of the mucopolysaccharidosis I (MPS I) patient to improve outcome assessments: evidence from the literature and treatment experts
  • Cognitive debriefing interviews on the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ)
  • Evaluating the MPS-HAQ for use in patient registries to support EMA regulatory commitments