Ultragenyx Clinical Survey in progress
Ultragenyx have started a GSD3 trial in the USA (not for UK at the moment).
The title of the study is ‘Clinical Survey Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III).’ It started in November 2020 and is due to complete in June 2021.
Natural History Study
This is a Natural History Study for GSD3, which is very important for information gathering.
Natural History Studies do not have a trial treatment but collate information about the course of a disease and also about what measures are good tests of improvements or deterioration in the condition. These measures can then be used later to test out new treatments.