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Registry of Patients with Lysosomal Acid Lipase Deficiency
This study is now recruiting.
This is an observational registry study intended to document the course of Lysosomal Acid Lipase Deficiency (LALD), in order to further understand the disease, its progression, which treatments are effective, and any related complications. As an observational study, no additional study visits - outside of subjects usual Metabolism clinic visit - are required. Eligible patients will be asked to sign an informed consent form prior to enrollment and will periodically have the option of completing questionnaires about their quality of life.
Who Do I Contact?
If you are interested in participating in the study or want to learn more please contact our study team at MetabolismResearch [at] chop.edu or 267-426-1368.
ClinicalTrials.gov Identifier
Visit Criteria
No visits outside of your usual clinic visits are required. Main study procedures include optional blood samples and medical record review.
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