Sebelipase alfa enzyme replacement therapy in Wolman disease: a nationwide cohort with up to ten years of follow-up

BACKGROUND: Wolman disease (WD), the rapidly progressive phenotype of lysosomal acid lipase (LAL) deficiency, presents in neonates with failure to thrive and hepatosplenomegaly, and leads to multi-organ failure and death before 12 months of age. In clinical trials, enzyme replacement therapy (ERT) w...

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Detalles Bibliográficos
Autores principales: Demaret, Tanguy, Lacaille, Florence, Wicker, Camille, Arnoux, Jean-Baptiste, Bouchereau, Juliette, Belloche, Claire, Gitiaux, Cyril, Grevent, David, Broissand, Christine, Adjaoud, Dalila, Abi Warde, Marie-Thérèse, Plantaz, Dominique, Bekri, Soumeya, de Lonlay, Pascale, Brassier, Anaïs
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2021
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8670257/
https://www.ncbi.nlm.nih.gov/pubmed/34906190
http://dx.doi.org/10.1186/s13023-021-02134-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8670257/
https://www.ncbi.nlm.nih.gov/pubmed/34906190
http://dx.doi.org/10.1186/s13023-021-02134-3

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