A Multicenter, Interventional, Retrospective and Prospective study of Enzyme Replacement Therapy (VPRIV) Clinical Outcomes and Safety in Gaucher Disease Type 1 Patients Previously Treated with Substrate Reduction Therapy
Study Description
Adult patients with Gaucher Disease Type 1 who have previously received SRT (substrate replacement therapy) and are now receiving ERT (enzyme replacement therapy) with velaglucerase alfa. For group B, no visits are required since this is a retrospective data collection. For group A, there are visits every 3 months for 1 year. These will include drawing blood, using an app on your cell phone to answer questionnaires, and an interview at the end of the study.
Eligibility Summary
Adult patients with Gaucher Disease Type 1 who have previously received SRT (substrate replacement therapy) and are now receiving ERT (enzyme replacement therapy) with velaglucerase alfa.
Investigators
- Estrella Lizbeth Mellin Sanchez, M.D.
Pediatrics