Alexion’s Kanuma (sebelipase alfa), which has been specifically recommended for use in patients who are aged two years or younger when administration begins, will now become the first treatment available on the NHS for the rapidly-progressive rare genetic disease.
Occurring in around one in 350,000 births, Wolman disease causes a build-up of fat in cells in the liver, heart, blood vessels and digestive system.
Symptoms in infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhoea, developmental delay and anaemia…
