A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-Care Setting
study id #: NCT05073783
condition: Pompe Disease, Mucopolysaccharidosis Type I (MPS I)
status: Not yet recruiting
purpose:Primary Objective:
To obtain data pertaining to the safety and tolerability of alglucosidase alfa and laronidase treatments administered in a home-care infusion setting.
Secondary Objectives:
To evaluate personal satisfaction of both cohorts of patients treated in a home-care infusion setting.
To evaluate the infusion compliance in both cohorts of patients treated in a home-care infusion setting.
results: https://clinicaltrials.gov/ct2/show/results/NCT05073783
last updated: February 26, 2022
-
Mucopolysaccharidoses Diagnosis in the Era of Enzyme Replacement Therapy in EgyptUndegraded glycosaminoglycans (GAGs) ind...
-
Fatal Care Research FoundationFatal Care Research Center inaugurated a...
-
Extraordinary Experiences ProgramProgram provides grants of up to $1000 t...
-
Deep Intronic Variant in the ARSB Gene as the Genetic Cause for Maroteaux-Lamy Syndrome (MPS VI)Maroteaux-Lamy syndrome (MPS-VI) is a ra...
-
Mucopolysaccharidosis Patients Have Reduced Functional CapacityMucopolysaccharidoses (MPSs) are a group...
-
Advocacy for MPS and MLAdvocacy is the best way for the MPS and...
-
Corneal Cloudiness: A Presenting Feature of Mucopolysaccharidosis Type IA 10-y-old ‘short-statured’ male pre...