Inborn Errors of Metabolism and Osteopetrosis

Robert Wynn; Ansgar Schulz

doi:10.1007/978-3-030-02278-5_90

Inborn Errors of Metabolism and Osteopetrosis

Review

In: The EBMT Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies [Internet]. 7th edition. Cham (CH): Springer; 2019. Chapter 90.

Authors

Robert Wynn¹, Ansgar Schulz²

Book Editors

Enric Carreras¹, Carlo Dufour², Mohamad Mohty³, Nicolaus Kröger⁴

Affiliations

¹ Blood and Marrow Transplant Unit, Royal Manchester Children’s Hospital, University of Manchester, Manchester, UK
² Department of Pediatrics, University Medical Center Ulm, Ulm, Germany

Book Affiliations

¹ Spanish Bone Marrow Donor Registry, Josep Carreras Foundation and Leukemia Research Institute, Catalunya, Barcelona, Spain
² Hematology Unit, IRCCS Giannina Gaslini, Geneva, Switzerland
³ Department of Hematology, Pierre and Marie Curie University, Paris, France
⁴ Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppend, Hamburg, Germany

Excerpt

Inborn errors of metabolism (IEM) comprise a large group of inherited disease, some of which are due to disordered lysosomal, peroxisomal, or mitochondrial function and only some of which might be improved following HSCT.

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Review