First-ever therapy for rare genetic disease in babies to save lives on the NHS

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. Following […]

Read More  on the NHS website.