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Pompe Disease Registry

The objectives of the Registry are: To enhance the understanding of the variability, progression, and natural history of the key manifestations of Pompe disease; To assist the Pompe medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care; To characterize …

Mucopolysaccharidosis I (MPS I) Registry

The data collected by the MPS I Registry will provide information to better characterize the natural history and progression of MPS I as well as the clinical responses of patients receiving enzyme replacement therapy, such as Aldurazyme (Recombinant Human Alpha-L-Iduronidase), or other treatment modalities. The objectives of the Registry are: …

Fabry Disease Registry & Pregnancy Sub-registry

The primary objectives of the Registry are: To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; To assist the Fabry medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient …