Farber's Lipogranulomatosis: Multimodal Therapy With Tocilizumab and Consolidative HSCT Improves Assessment, and Long‐Term Outcome

ABSTRACT Farber's lipogranulomatosis (FL) is an autosomal recessive lipid storage disorder, arising as a consequence of genetic acid ceramidase deficiency. Clinically, it presents as severe arthritis, voice hoarseness, and widespread, painful subcutaneous nodules (SCN). For those without CNS involve...

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Bibliografiske detaljer

Principais autores:	Nathanael C. C. Lucas, Claire Horgan, Omima Mustafa, Srividhya Senthil, Denise Bonney, Ramya Nataraj, Sophie Fisher, Chern Tan, Stewart Rust, Simon A. Jones, Sarah Hulley, Robert Wynn
Format:	Artigo
Sprog:	Inglês
Udgivet:	Wiley 2025-09-01
Serier:	JIMD Reports
Fag:	Farber's lipogranulomatosis haematopoietic stem cell transplant subcutaneous nodules tocilizumab
Online adgang:	https://doi.org/10.1002/jmd2.70041
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