The Global Hypophosphatasia Registry: lessons learned from a decade of real-world data

Abstract excerpt

INTRODUCTION: Hypophosphatasia (HPP) is an inherited, metabolic, rare disease characterized by a high level of clinical heterogeneity. In response to this robust heterogeneity, the Global HPP Registry was formed to characterize the types of manifestations that patients may experience, as well as to compile information on genetic underpinnings of the disease, overall impact on patient quality of life, and safety...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Alkaline Phosphatase Research Studies
• Share to Bone health and osteoporosis research
• Share to Bone health and treatments