Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia-Reference-Cited by-同舟云学术

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Five-year efficacy and safety of asfotase alfa therapy for adults and adolescents with hypophosphatasia

Published:2019-04 Issue: Volume:121 Page:149-162
ISSN:8756-3282
Container-title:Bone
language:en
Short-container-title:Bone

Author:

Kishnani Priya S.,Rockman-Greenberg Cheryl,Rauch Frank,Bhatti M. Tariq,Moseley Scott,Denker Andrew E.,Watsky Eric,Whyte Michael P.

Funder

Alexion Pharmaceuticals

Publisher

Elsevier BV

Subject

Histology,Physiology,Endocrinology, Diabetes and Metabolism

Reference47 articles.

1. Hypophosphatasia: an overview for 2017;Whyte;Bone,2017

2. A missense mutation in the human liver/bone/kidney alkaline phosphatase gene causing a lethal form of hypophosphatasia;Weiss;Proc. Natl. Acad. Sci. U. S. A.,1988

3. Genetic analysis of adults heterozygous for ALPL mutations;Taillandier;J. Bone Miner. Metab.,2018

4. Different missense mutations at the tissue-nonspecific alkaline phosphatase gene locus in autosomal recessively inherited forms of mild and severe hypophosphatasia;Henthorn;Proc. Natl. Acad. Sci. U. S. A.,1992

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1. Efficacy and safety of asfotase alfa in patients with hypophosphatasia: A systematic review;Bone;2024-11

2. Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia;Journal of Bone and Mineral Research;2024-08-13

3. Longitudinal course of circulating miRNAs in a patient with hypophosphatasia and asfotase alfa treatment: a case report;JBMR Plus;2024-08-10

4. Challenges in Hypophosphatasia: Suspicion, Diagnosis, Genetics, Management, and Follow-Up;Hormone Research in Paediatrics;2024-08-05

5. Integrated Applied Clinical Pharmacology in the Advancement of Rare and Ultra‐Rare Disease Therapeutics;Clinical Pharmacology & Therapeutics;2024-07-22

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