"Patients with Wolman disease typically present during the first two months of life with failure to thrive, diarrhoea and vomiting. Abdominal distention may be present because of hepatomegaly, splenomegaly and generalised lymphadenopathy 2."
"Patients with Wolman disease typically present during the first two months of life with failure to thrive, diarrhoea and vomiting. Abdominal distention may be present because of hepatomegaly, splenomegaly and generalised lymphadenopathy 2."
"Historically, treatment approaches have included total parenteral nutrition (TPN), corticosteroids, plasma infusion, and various dietary supplements, all without success, with death in infancy ensuing (usually within 6 months). Bone marrow transplantation has been reported with some success 6. Enzyme replacement therapy with sebelipase alfa has also increased survival and decreased morbidity 7."