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Long-term survival with sebelipase alfa enzyme replacement therapy in infants with rapidly progressive lysosomal acid lipase deficiency: final results from 2 open-label studies
BACKGROUND: If symptomatic in infants, the autosomal recessive disease lysosomal acid lipase deficiency (LAL-D; sometimes called Wolman disease or LAL-D/Wolman phenotype) is characterized by complete loss of LAL enzyme activity. This very rare, rapidly progressive form of LAL-D results in severe man...
Autores principales: | Vijay, Suresh, Brassier, Anais, Ghosh, Arunabha, Fecarotta, Simona, Abel, Florian, Marulkar, Sachin, Jones, Simon A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7789691/ https://www.ncbi.nlm.nih.gov/pubmed/33407676 http://dx.doi.org/10.1186/s13023-020-01577-4 |
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