Treatment for Alagille Syndrome Granted Orphan Drug Designation
Alagille syndrome affects approximately 1 in 70,000 newborns.
All articles by Da Hee Han, PharmD
RET Inhibitor Gets Breakthrough Tx Status for Medullary Thyroid Cancer
LOXO-292 is being evaluated in the ongoing Phase 1/2 LIBRETTO-001 clinical trial.
Kanuma Approved for Lysosomal Acid Lipase Deficiency
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).
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