Matza, Louis S.; Stewart, Katie D.; Fournier, Marie; … - In: The European Journal of Health Economics 25 (2024) 8, pp. 1437-1448
Introduction: Acid sphingomyelinase deficiency (ASMD) type B is a rare genetic disorder leading to enlargement of the spleen and liver, pulmonary dysfunction, and other symptoms. Cost-utility analyses are often conducted to quantify the value of new treatments, and these analyses require health...