deficiency

(1.) Htmes RW, Barlow SE, Bove K, Qutntantlh NM, Sheridan R, Kohh R Lysosomal Acid Lipase Deficiency Unmasked in Two Children with Nonalcoholic Fatty Liver Disease Pediatrics 2016, 138.

Alexion also has two innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia and lysosomal acid lipase deficiency (LAL-D).

Survival in infants treated with sebelipase alfa for lysosomal acid lipase deficiency: an open-label, multicenter, dose-escalation study.

Ala, "Wolman's disease and cholesteryl ester storage disorder: the phenotypic spectrum of lysosomal acid lipase deficiency," The Lancet Gastroenterology and Hepatology, vol.

The remaining 38 agents can be classified into the following categories: anticoagulant (2), antidiarrheal (1), antidiabetic (1), antidote (3), antiemetic (1), antifungal (1), antilipemic (2), antineoplastic (13), antipsychotic (2), antiviral (1), bile acid (2), cardiovascular (2), female sexual dysfunction (1), immunomodulator (1), lysosomal acid lipase deficiency (1), parathyroid hormone (1), pyrimidine analog (1), and respiratory (2).