Lysosomal acid lipase deficiency (LAL-D) is rare, with a reported incidence of 1 in 40,000 to 300,000. These mutations in the ...

Alexion has gained FDA approval for Kanuma, its treatment for the rare disease lysosomal acid lipase (LAL) deficiency. Alexion says Kanuma (sebelipase alfa) could eventually bring in more than $ ...

Inaaya, from Sheldon, Birmingham, was born with a rare genetic condition called Wolman's Disease, a type of lysosomal acid lipase deficiency that can cause multi-organ damage in infants.