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Registry of Patients with Lysosomal Acid Lipase Deficiency

Registry of Patients with Lysosomal Acid Lipase Deficiency

Contact

If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is now recruiting. Learn more about enrolling here.

Description

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. 

What to expect

No visits outside of your usual clinic visits are required. Main study procedures include optional blood samples and medical record review.
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.

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