In a striking revelation in pediatric medicine, a recent case report has highlighted neonatal hypophosphatasia, an exceptionally rare genetic disorder that

In a significant development for the treatment of hypophosphatasia (HPP), groundbreaking research has emerged detailing the effectiveness of Asfotase Alfa—a recombinant form of tissue-nonspecific alkaline phosphatase—ten years after its approval. This study, authored by Khan, A.A., Rush, E.T., Wakeford, C., and others, compiles critical insights from both clinical research and real-world evidence, providing a comprehensive overview of Asfotase Alfa's impact on patient outcomes in the long term.

In a striking revelation in pediatric medicine, a recent case report has highlighted neonatal hypophosphatasia, an exceptionally rare genetic disorder that

Vicky E MacRae; ENPP1 Inhibition: A Promising Oral Therapy for Later-Onset Hypophosphatasia, Journal of Bone and Mineral Research, , zjaf154, https://doi.o

Abstract. We previously documented successful resolution of skeletal and dental disease in the infantile and late-onset murine models of hypophosphatasia (

New study reveals promising gene therapy for Hypophosphatasia, potentially replacing invasive treatments with a single injection for lifelong benefits.

He spent several weeks digging into his medical records and scrolling through scientific websites before hitting pay dirt.