Mucopolysaccharidosis II Clinical Trial
Official title:
Registry of Patients Diagnosed With Lysosomal Storage Diseases
This is an international prospective and retrospective registry of patients with Lysosomal Storage Diseases (LSDs) to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with LSDs.
The need for methods to track patient outcomes, clinical management, medical decision making, and quality of care are all part of current national mandates in patient safety and quality of care delivery. The aim of this registry is to prospectively and retrospectively collect data on patients who are diagnosed with Lysosomal Storage Disease and other LSD mutations. Data collected will be used to: 1. Identify patient outcomes of therapies. 2. Improve clinical management of patients with LSDs. 3. Improve medical decision making. 4. Improve quality of care. ;
Status | Clinical Trial | Phase | |
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Withdrawn |
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