Life-saving treatment for babies with rare disease rolled out on NHS

Press/Media: Research


Babies and toddlers with a rare and fatal genetic condition can now receive a life-saving treatment on the NHS for the first time.

Sebelipase alfa (Kanuma®), an enzyme replacement therapy, will become the first treatment available on the NHS for Wolman disease, a condition which presents in babies and children under two years old.

Period27 Nov 2023

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