Efficacy and Safety of Asfotase Alfa in Infants and Young Children With Hypophosphatasia: A Phase 2 Open-Label Study

Author:

Hofmann Christine E1ORCID,Harmatz Paul2,Vockley Jerry34,Högler Wolfgang56,Nakayama Hideki7,Bishop Nick89,Martos-Moreno Gabriel Á101112,Moseley Scott13,Fujita Kenji P13,Liese Johannes14,Rockman-Greenberg Cheryl1516,

Affiliation:

1. University Children’s Hospital, University of Würzburg, Würzburg, Germany

2. University of California San Francisco Benioff Children’s Hospital Oakland, Oakland, California

3. University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania

4. University of Pittsburgh, Graduate School of Public Health, Pittsburgh, Pennsylvania

5. Department of Paediatrics and Adolescent Medicine, Johannes Kepler University, Linz, Austria

6. Institute of Metabolism and Systems Research, University of Birmingham, Birmingham, United Kingdom

7. Fukuoka Higashi Medical Center, Fukuoka, Japan

8. Sheffield Children’s Hospital, Sheffield, United Kingdom

9. University of Sheffield, Sheffield, United Kingdom

10. Hospital Infantil Universitario Niño Jesús, IIS La Princesa, Madrid, Spain

11. Universidad Autónoma de Madrid, Madrid, Spain

12. Centro de Investigación Biomédica en Red de la Fisiopatología de la Obesidad y Nutrición, Instituto de Salud Carlos III, Madrid, Spain

13. Alexion Pharmaceuticals, Inc., Boston, Massachusetts

14. Department of Pediatrics, University Hospital of Würzburg, Würzburg, Germany

15. Children’s Hospital Research Institute of Manitoba, Winnipeg, Manitoba, Canada

16. University of Manitoba, Winnipeg, Manitoba, Canada

Funder

Alexion Pharmaceuticals, Inc.

National Center for Advancing Translational Sciences

National Institutes of Health

Publisher

The Endocrine Society

Subject

Biochemistry (medical),Clinical Biochemistry,Endocrinology,Biochemistry,Endocrinology, Diabetes and Metabolism

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