Posted by Michael Wonder on 27 Nov 2023
Lifeline for Wolman disease kids as NICE backs Alexion drug
27 November 2023 - Infants with a rare and fatal congenital disorder called Wolman disease in England and Wales will soon have access to a new drug via the NHS that can dramatically extend their lives.
NICE has published final draft guidance recommending Kanuma (sebelipase alfa) – sold by AstraZeneca’s Alexion unit – after the company reached a commercial deal with NHS England.
