First treatment recommended for infants with Wolman Disease

Retrieved on: 2023-11-27 20:27:39

Tags for this article:

Lipid storage disorders

Rare diseases

Lysosomal acid lipase deficiency

Kanuma

Enzyme replacement therapy

Wolman

National Institute for Health and Care Excellence

Inborn errors of metabolism

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First treatment recommended for infants with Wolman Disease. View article details on HISWAI: https://www.europeanpharmaceuticalreview.com/news/189307/first-treatment-recommended-for-infants-for-wolman-disease/

Excerpt

Alexion's intravenous enzyme replacement therapy has been recommended for the ultra-rare disease by the NICE.

Article found on: www.europeanpharmaceuticalreview.com

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