Geneva, June 22 -- International Clinical Trials Registry received information related to the study (ISRCTN15729514) titled 'Using advanced MRI scans and muscle function tests to study muscle changes in adults with Pompe disease receiving enzyme replacement therapy' on June 15.
Study Type: Observational
Study Design:
Cohort study
Primary Sponsor: Newcastle upon Tyne Hospitals NHS Foundation Trust
Condition:
Pompe disease
Nutritional, Metabolic, Endocrine
Intervention:
PRISM is a single-centre, prospective, longitudinal, observational cohort study conducted in the JWMDRC, in Newcastle upon Tyne, UK.
The study recruits 40 adult participants: 30 adults with late-onset Pompe disease (LOPD) who are already receiving licensed enzyme r...