Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patients

Mucopolysaccharidosis type I-H (MPS I-H) is a rare lysosomal storage disorder caused by α-L-Iduronidase deficiency. Early haematopoietic stem cell transplantation (HSCT) is the sole available therapeutic option to preserve neurocognitive functions. We report long-term follow-up (median 9 years, inte...

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Detalles Bibliográficos
Autores principales: Gardin, Antoine, Castelle, Martin, Pichard, Samia, Cano, Aline, Chabrol, Brigitte, Piarroux, Julie, Roubertie, Agathe, Nadjar, Yann, Guemann, Anne-Sophie, Tardieu, Marine, Lacombe, Didier, Robert, Matthieu P., Caillaud, Catherine, Froissart, Roseline, Leboeuf, Virginie, Barbier, Valérie, Bouchereau, Juliette, Schiff, Manuel, Fauroux, Brigitte, Thierry, Briac, Luscan, Romain, James, Syril, de Saint-Denis, Timothée, Pannier, Stéphanie, Gitiaux, Cyril, Vergnaud, Estelle, Boddaert, Nathalie, Lascourreges, Claire, Lemoine, Michel, Bonnet, Damien, Blanche, Stéphane, Dalle, Jean-Hugues, Neven, Bénédicte, de Lonlay, Pascale, Brassier, Anaïs
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10005963/
https://www.ncbi.nlm.nih.gov/pubmed/36494569
http://dx.doi.org/10.1038/s41409-022-01886-1

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