Adnkronos "A drug, an enzyme therapy, is available that replaces the enzyme deficiency caused by this disease. Sebelipase is a lifesaving drug in neonatal and pediatric forms, where survival is impossible without treatment. However, with treatment, the early onset of the disease is treated, with long-term survival confirmed so far." These were the words of Lorenzo D'Antiga, director of the maternal, infant, and pediatric department at the Papa Giovanni XXIII Hospital in Bergamo, speaking today in Milan at a press conference organized by Alexion, a biopharmaceutical company of the AstraZeneca Group, for the Italian launch of sebelipase alfa, an enzyme replacement therapy for the treatment of patients of all ages with lysosomal acid lipase deficiency (LAL-d).
https://www.youtube.com/watch?v=0cBlbipyGEE