The cause of Niemann-Pick disease is relatively complicated. People who have this disease inherit one of ... Xenpozyme is an enzyme replacement therapy approved by the Food and Drug Administration ...

Niemann-Pick disease affects multiple systems throughout ... It's also common for swallowing to cause pain, and for people to feel like food is stuck in the chest or throat. Some individuals ...

Niemann-Pick disease type C (NPC) has been reported in association with inflammatory bowel disease. In cases where colitis has been reported in association with NPC, the neurological manifestations of ...

In the space of four days, the number of FDA-approved therapies for ultra-rare genetic disorder Niemann-Pick disease has gone ... that there are a few hundred people in the US living with NPC.

The cause of Niemann-Pick disease is relatively complicated. People who have this disease ... is an enzyme replacement therapy approved by the Food and Drug Administration (FDA) for the treatment ...