An Observational Longitudinal Prospective Long-Term Registry of Patients With Hypophosphatasia

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An Observational Longitudinal Prospective Long-Term Registry of Patients With Hypophosphatasia

Brief description of study

This multinational, multicenter, observational, prospective, long-term registry is designed to collect data on epidemiology, hypophosphatasia (HPP) history, clinical course, symptoms (including systemic aspects of disease), and burden of disease from patients of all ages who have a diagnosis of HPP. In addition, the Registry will collect data on asfotase alfa dosing, effectiveness of treatment, serious adverse events (SAEs), antidrug antibodies (ADAs) against asfotase alfa, pregnancy and neonatal outcome data (for patients treated with asfotase alfa only), and targeted events.

Clinical Study Identifier: s22-00229

Principal Investigator: Berrin Monteleone.

Other Investigator: Liana Gabriel.

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