PARLIAMENTARY WRITTEN QUESTION
(29 August 2025)

The Government is committed to improving the lives of those living with rare diseases, such as Niemann-Pick disease type B. The UK Rare Diseases Framework sets out four priorities collaboratively developed with the rare disease community. These include: helping patients get a final diagnosis faster; increasing awareness of rare diseases among healthcare professionals; better coordination of care; and improving access to specialist care, treatments, and drugs. We published the annual England Rare Diseases Action Plan in February 2025, where we report on the steps we have taken to advance these priorities.