Meghann Ferguson, Director-at-Large at the National Niemann-Pick Disease Foundation and mother of a 7-year-old boy with Niemann-Pick Disease Type C (NPC), talks about the current crisis NPC families are facing as adrabetadex, an experimental drug being evaluated as a treatment for NPC, is scheduled to be discontinued.

NPC is a disabling neurogenetic disorder that has been diagnosed prenatally, neonatally, during childhood, and even into adulthood. This very rare genetic disorder is marked by progressive motor dysfunction and a highly variable symptom profile and onset of symptoms. It can result in the patient’s death soon after birth or manifest as a chronic disorder with symptoms worsening slowly over time.

Ms. Ferguson explains that her son is one of many patients currently on adrabetadex who has shown improvement. Despite this, it has been announced that adrabetadex will be discontinued in October. Ms. Ferguson and other NPC parents and physicians are fighting to keep this drug on the market. 

To learn more about Niemann-Pick disease and other rare neurological disorders, visit