Rare Diseases: Children

(asked on 22nd July 2020) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps he is taking to ensure access to enzyme replacement treatment at a local specialist centre for children with rare diseases.


Answered by
Jo Churchill Portrait
Jo Churchill
Minister of State (Department for Work and Pensions)
This question was answered on 1st September 2020

The Government is committed to improving the lives of those affected by rare disease and continues to implement the commitments made in the UK Strategy for Rare Diseases.

There are three specialist centres in the country that prescribe enzyme replacement therapy for the treatment of lysosomal storage disorders. These diseases are very rare and the expertise to treat this cohort of patients is concentrated in these centres where staff are experienced in their care and where they are also engaged in research into these conditions.

Enzyme replacement therapy is usually delivered via an infusion at home with the support of homecare providers.

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