Enzyme-Replacement Therapy in Life-Threatening Hypophosphatasia

2012559 citationsJournal Articlebronze Open Access

Authors

Michael P. Whyte · Shriners Hospitals for Children - Erie

Cheryl R. Greenberg

Nada Salman · Tawam Hospital

Michael B. Bober · Alfred I. duPont Hospital for Children

William H. McAlister · St. Louis Children's Hospital

Deborah Wenkert · Shriners Hospitals for Children - Erie

Bradley J. Van Sickle · Monroe Carell Jr. Children's Hospital

Abstract

ENB-0040, an enzyme-replacement therapy, was associated with improved findings on skeletal radiographs and improved pulmonary and physical function in infants and young children with life-threatening hypophosphatasia. (Funded by Enobia Pharma and Shriners Hospitals for Children; ClinicalTrials.gov number, NCT00744042.).

Topics & Keywords

Alkaline Phosphatase Research Studies Bone health and osteoporosis research Vitamin D Research Studies

UN Sustainable Development Goals

Good health and well-being

Publication Details

Published in: New England Journal of Medicine

Volume 366, Issue 10, pp. 904-913

DOI: 10.1056/nejmoa1106173

Field-Weighted Citation Impact: 23.79