Santiago de Compostela University Researchers Update Current Data on Wolman Disease [Twice weekly dosing with Sebelipase alfa (Kanuma(R)) rescues severely ill infants with Wolman disease].

A recent study conducted by researchers at Santiago de Compostela University has found that a twice-weekly dosing regimen of Sebelipase alfa (Kanuma) effectively treats severely ill infants with Wolman disease. The study evaluated the pharmacokinetics and pharmacodynamics of Sebelipase alfa at a dosage of 5 mg/kg twice weekly, which had not been previously assessed in clinical trials. The results showed that this intensive regimen increased substrate clearance and was well tolerated by patients. However, there was still lipid accumulation between doses. This research provides valuable insights into the treatment of Wolman disease and offers hope for improved outcomes for patients. [Extracted from the article]