Sanfilippo syndrome

  • Payer research into a new treatment for mucopolysaccharidosis III (San Filippo syndrome)
  • Preliminary consulting on endpoints in San Filippo syndrome
  • Endpoint review and caregiver interviews in San Filippo syndrome
  • Participation in a meeting to provide consultation on a paediatric PRO in San Filippo syndrome
  • Strategic PRO and ObsRO analysis of a San Filippo natural history study database