Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia

Margaret Robb November 12, 2024

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Categories: Scientific Publications, Scientific Publications (new)

Tags: Adeno-associated virus vector, alkaline phosphatase, Genetic Therapies, hypophosphatasia, Progress, therapeutics

A Family’s Fight Against HPP

On December 21, 2011 our daughter Clara B. was born; at the time we believed she was a beautiful healthy baby girl. Little did we…

Taralyn Naslonski November 6, 2020

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Letter to the Editor: “Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study”

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softbones October 28, 2020

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Pharmacokinetics of Asfotase Alfa in Adult Patients With Pediatric‐Onset Hypophosphatasia

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Denise Goodbar April 9, 2021

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Detection of hypophosphatasia in hospitalised adults in rheumatology and internal medicine departments: a multicentre study over 10 years

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Margaret Robb August 15, 2024

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Five-Year Efficacy and Safety of Asfotase Alfa Therapy for Adults and Adolescents with Hypophosphatasia

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softbones October 28, 2020

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