Overview of pediatric and adult lysosomal acid lipase deficiency: expert recommendations from a Gulf cooperation council working group
Orphanet J Rare Dis. 2025 Dec 5. doi: 10.1186/s13023-025-04158-5. Online ahead of print. ABSTRACT BACKGROUND: Lysosomal acid lipase deficiency (LAL-D) is an autosomal recessive ultrarare …