High-Dose ERT, Rituximab, and Early HSCT in an Infant with Wolman’s Disease-Reference-Cited by-同舟云学术

High-Dose ERT, Rituximab, and Early HSCT in an Infant with Wolman’s Disease

Published:2024-02-15 Issue:7 Volume:390 Page:623-629
ISSN:0028-4793
Container-title:New England Journal of Medicine
language:en
Short-container-title:N Engl J Med

Author:

Eskandari Siawosh K.¹^ORCID,Revenich Elisabeth G.M.¹,Pot Dirk J.¹,de Boer Foekje¹,Bierings Marc¹,van Spronsen Francjan J.¹,van Hasselt Peter M.¹,Lindemans Caroline A.¹,Lubout Charlotte M.A.¹

Affiliation:

1. From the Department of Metabolic Diseases, Beatrix Children’s Hospital (S.K.E., F.B., F.J.S., C.M.A.L.), and the Department of Surgery (S.K.E.), University Medical Center (UMC) Groningen, Groningen, the Division of Pediatrics (E.G.M.R., M.B., P.M.H., C.A.L.) and the Department of Metabolic Diseases (P.M.H.), UMC Utrecht, and the Department of Stem Cell Transplantation, Princess Máxima Center for Pediatric Oncology (E.G.M.R., M.B., C.A.L.), Utrecht, and the Department of Pediatrics, Gelre Hospital,...

Publisher

Massachusetts Medical Society

Link

http://www.nejm.org/doi/pdf/10.1056/NEJMoa2313398

Reference22 articles.

1. Wolman's disease and cholesteryl ester storage disorder: the phenotypic spectrum of lysosomal acid lipase deficiency

2. Rapid progression and mortality of lysosomal acid lipase deficiency presenting in infants

3. Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman disease

4. Long-Term Sebelipase Alfa Treatment in Children and Adults With Lysosomal Acid Lipase Deficiency

5. Survival in infants treated with sebelipase Alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study

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