Adnkronos The Italian Medicines Agency (AIFA) has approved the reimbursement of sebelipase alfa in Italy for the treatment of lysosomal acid lipase deficiency (LAL-D). This long-term enzyme replacement therapy is indicated for patients of all ages and is the first treatment available in the country for this ultra-rare and progressive genetic metabolic disease caused by a defect in the enzyme that metabolizes cholesterol and triglycerides.
This failure leads to the accumulation of lipids in the liver and other organs, causing various damage. According to experts, sebelipase alfa "represents a life-saving enzyme therapy in neonatal and pediatric patients." This was discussed during a press conference organized by Alexion, a biopharmaceutical company part of the AstraZeneca group.
https://www.youtube.com/watch?v=aaNvmvhg9u4