Clinical Trial: Mucolipidosis Type IV Natural History Study
Study Status: Completed
Recruit Status: RECRUITING
Condition: Mucolipidosis Type IV
Study Type: OBSERVATIONAL
Official Title: A New Retrospective Natural History Study of Mucolipidosis Type IV
Brief Summary:
The primary objectives of the study are: to describe the characteristics of the current international MLIV population; to define the median age at which patients with MLIV achieve or lose developmental milestones; to define the natural history of MLIV for the Gross Motor Function Classification System (GMFCS) (Morris and Bartlett, 2004) and the MLIV specific scale and test the validity of retrospectively applying these scales to medical record data; to define the rate of visual decline in patients with MLIV.
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