Status In progress
Process HST
ID number 800

Project Team

Project lead Joanne Ekeledo

Email enquiries


Companies sponsors Chiesi Ltd
Others Addenbrooke's Lysosomal Disorders Unit
  Birmingham Children’s Hospital NHS Foundation Trust Lysosomal Storage Disorders Unit
  Department of Health
  National Hospital for Neurology and Neurosurgery Charles Dent Metabolic Unit
  NHS England
  Royal Free Lysosomal Storage Disorders Unit
  Willink Unit, Genetic Medicine, Central Manchester Foundation Trust
Patient carer groups MPS Society
Professional groups Royal College of Physicians
  Royal College of Paediatrics and Child Health


Comparator companies None
General commentators All Wales Therapeutics and Toxicology Centre
  British National Formulary
  Department of Health, Social Services and Public Safety for Northern Ireland
  Healthcare Improvement Scotland
  Welsh Government
  Welsh Health Specialised Services Committee


Key events during the development of the guidance:

Date Update
28 June 2018 Committee meeting: 2
23 May 2018 - 14 June 2018 Evaluation consultation: 1
25 April 2018 Committee meeting: 1
08 November 2017 Invitation to participate
20 March 2017 Referral
20 March 2017 In progress, Topic referred.
22 September 2016 - 21 October 2016 Consultation on suggested remit, draft scope and provisional matrix of consultees and commentators

For further information on how we develop guidance, please see our page about NICE highly specialised technologies guidance