Treatment for Alagille Syndrome Granted Orphan Drug Designation
Alagille syndrome affects approximately 1 in 70,000 newborns.
Alagille syndrome affects approximately 1 in 70,000 newborns.
LOXO-292 is being evaluated in the ongoing Phase 1/2 LIBRETTO-001 clinical trial.
Alexion announced that the Food and Drug Administration (FDA) has approved Kanuma (sebelipase alfa) for the treatment of patients with lysosomal acid lipase deficiency (LAL-D).