250
Participants
Start Date
January 1, 2014
Primary Completion Date
September 30, 2028
Study Completion Date
December 31, 2028
Stratified response to Enzyme Therapy
Observational study involves review of retrospective and prospective data of participants' medical history, pathology, imaging and health questionnaires.
TERMINATED
Birmingham Childrens Hospital, Birmingham
RECRUITING
New Queen Elizabeth Hospital, Birmingham
RECRUITING
Cambridge University Hospital, Cambridge
RECRUITING
Great Ormond Street Hospital, London
RECRUITING
National Hospital for Neurology and Neurosurgery, London
RECRUITING
Royal Free Hospital, London
RECRUITING
Royal Manchester Childrens Hospital, Manchester
RECRUITING
Salford Royal NHS Foundation Trust, Salford
Collaborators (1)
Medical Research Council
OTHER_GOV
National Institute for Health Research, United Kingdom
OTHER_GOV
Cambridge University Hospitals NHS Foundation Trust
OTHER