Lysosomal Acid Lipase Deficiency (LAL-D) in Infants

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Nearly 90% mortality within 12 months after birth in infants with LAL-D¹

^aA retrospective chart review and data extraction of 35 patients diagnosed with LAL-D before age 2 (26 with growth failure before 6 months of life, 9 without).¹

^bAlthough the protocol allowed for enrollment of living patients, all patients in the overall population were deceased.¹

43

of infant patients with LAL-D experience symptom onset before 1 month of age¹^,ii

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Footnotes:

ⁱEarly growth failure was defined as fulfillment of 1 or more of the following criteria before 6 months of age: (1) decreased body weight across ≥2 of the 11 major percentiles on a standard World Health Organization (WHO) weight-for-age chart; (2) body weight below the tenth percentile on a standard WHO weight-for-age chart and no weight gain within the previous 2 weeks; (3) loss of ≥5% of birth weight in children >2 weeks of age.¹

ⁱⁱFour patients survived beyond 12 months of age; of those, 2 underwent HSCT, 1 underwent liver transplant and HSCT, and 1 was untreated.¹

References: 1. Jones SA, et al. Genet Med. 2016;18:452-8. doi:10.1038/gim.2015.108.