Posted by Michael Wonder on 15 Jan 2024
Sebelipase alfa for the treatment of patients with Wolman disease (final guidance)
10 January 2023 - Sebelipase alfa is recommended as an option for the treatment of patients with Wolman disease only if treatment is started before 2 years of age.
It is recommended only if Alexion Pharmaceuticals provides sebelipase alfa according to the commercial arrangement.
