NHS first with baby therapy

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A treatment for a rare genetic condition that can kill babies before they turn one is to be available in England in a first for the NHS.

Wolman disease is a type of lysosomal acid lipase deficiency that occurs in one in every 350,000 births. The condition causes a build-up of fat in cells in the heart, blood vessels, liver and digestive system.

Sebelipase alfa, also known as Kanuma, is an enzyme replacement therapy given weekly via intravenous infusions. Prior to the therapy’s approval by the National Institute for Health and Care Excellence, there were no treatments.

The treatment will be delivered at Manchester University NHS Foundation Trust, Birmingham Women’s and Children’s Hospital and Great Ormond Street Hospital.

Medics said this “marks a major milestone”.

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