Niemann-Pick disease causes a variety of medical problems, and it often advances rapidly. The symptoms and effects of all of the variants of Niemann-Pick disease result from the buildup of ...

There are several markers that can indicate whether you have a heart problem. Like many conditions there are symptoms that the patient themselves will pick up on ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... to treat neurological symptoms in adults and ...