Mucopolysaccharidosis VII Disease Monitoring Program
study id #: NCT03604835
condition: Mucopolysaccharidosis VII, MPS VII, MPS 7, Sly Syndrome
status: Recruiting
purpose:The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.
intervention: No Intervention
results: https://clinicaltrials.gov/ct2/show/results/NCT03604835
last updated: February 26, 2022
-
Macular Changes in a Mucopolysaccharidosis Type I Patient With Earlier Systemic TherapiesTo describe retinal findings in a patien...
-
Mucopolysaccharidosis II (MPS II) in a Free-Living Kaka (Nestor Meridionalis) in New ZealandA lysosomal storage disease, identified ...
-
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...
-
A Review of the Clinical Outcomes in Idursulfase-Treated and Untreated Filipino Patients With Mucopolysaccharidosis ...Mucopolysaccharidosis type II (MPS II; H...
-
Awake Airway Endoscopy in Mucopolysaccharidosis: A Case ReportMucopolysaccharidosis (MPS) are a group ...
-
Heather Lau, MD, MSHeather Lau, M.D. M.S. is a board certif...
-
Issues of COVID-19-Related Distance Learning for Children With Neuronopathic MucopolysaccharidosesThe COVID-19 pandemic has impacted the e...