Correction to: Orphanet J Rare Dis (2021) 16:13 https://doi.org/10.1186/s13023-020-01577-4
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Plain Language Summary
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Lysosomal acid lipase deficiency (LAL-D) is a rare, inherited disease in which fatty material (cholesterol and triglycerides) becomes trapped in cells throughout the body, causing organ damage
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Infants can experience a particularly aggressive form of this disease where the functioning of the liver and intestine is impaired, thus leading to an enlarged abdomen and failure to grow and thrive
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If left untreated, LAL-D in infants leads to death, usually by 6 months of age
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This publication reports the results from 2 studies involving 19 infants with rapidly progressive LAL-D; infants received once-weekly intravenous infusions of sebelipase alfa for up to 3 or 5 years, depending on the study
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Results show that with sebelipase alfa treatment, the likelihood of an infant with LAL-D surviving to 12 months of age is 79% and the likelihood of surviving to 5 years of age is 68%
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Throughout both studies, treatment with sebelipase alfa was associated with (1) improvements in growth (weight, length/height, and arm circumference), (2) improvements in liver function, and (3) a decrease in liver and spleen size
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All patients experienced 1 or more adverse events (unwanted side effects), most of which were mild or moderate in severity; no patient stopped receiving treatment because of these events
Reference
Vijay S, et al. 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. Orphanet J Rare Dis. 2021;16:13. https://doi.org/10.1186/s13023-020-01577-4.
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Vijay, S., Brassier, A., Ghosh, A. et al. Correction to: 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. Orphanet J Rare Dis 16, 113 (2021). https://doi.org/10.1186/s13023-021-01753-0
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DOI: https://doi.org/10.1186/s13023-021-01753-0