The study is published in the journal Gastro Hep Advances. Lysosomal acid lipase (LAL) deficiency is a very rare genetic metabolic disorder caused by a deficiency in the lysomal acid lipase (LAL ...

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 $ ...