Posted by Michael Wonder on 15 May 2025
Deal agreed for continued access to life-changing treatment for rare inherited disease
14 May 2025 - NICE is consulting on draft guidance for cerliponase alfa for treating neuronal ceroid lipofuscinosis type 2, a type of Batten disease.
The draft guidance does not, at present, recommend the treatment’s use for future patients with this rare, life limiting disease due to its high price and the limited evidence of long-term effectiveness.
