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 ...

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 ...

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.