The deadly rare condition that can kill children before their first birthday

Families are raising awareness of a rare genetic condition that usually kills babies before they turn one. One child was diagnosed with the illness in a scan before he was born – after already having an older sibling die as a result of the disease.

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.

Symptoms include enlarged livers or spleens, poor weight gain and muscle tone, jaundice, anaemia, and developmental delays. Now, a treatment for the rare condition is to be made available in England in a first for the NHS, hoped to save the lives of toddlers and babies born with Wolman disease.

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Prior to the therapy’s approval by the National Institute for Health and Care Excellence (NICE), there were no treatments available for Wolman disease on the NHS, with the only options being palliative care or managing symptoms. Without treatment, babies with the condition usually do not live past their first birthday.

Eight-year-old Shoaib, from Halifax, was diagnosed with Wolman disease through a scan before he was born. He started the new treatment – named sebelipase alfa – at three days old after having an older sibling pass away due to the condition.

After being on the therapy for two years, Shoaib had a haematopoietic stem cell transplant and no longer requires treatment. Shoaib’s mother, Nadia, said: “When Shoaib was diagnosed with Wolman disease we were incredibly worried as we’d already lost another child to the illness.

“However, the