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The role of sebelipase alfa in the treatment of lysosomal acid lipase deficiency
Lysosomal acid lipase deficiency (LALD) is a lysosomal storage disorder (LSD) characterized either by infantile onset with fulminant clinical course and very poor prognosis or childhood/adult-onset disease with an attenuated phenotype. The disorder is often misdiagnosed or remains undiagnosed in chi...
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| Publicado no: | Therap Adv Gastroenterol |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
SAGE Publications
2017
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5484437/ https://ncbi.nlm.nih.gov/pubmed/28804516 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1756283X17705775 |
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