Niemann-Pick disease

Niemann-Pick disease is a rare genetic disorder where harmful fatty substances, called lipids, build up in cells, especially in the liver, spleen, brain, and other organs. This accumulation happens because of missing or malfunctioning enzymes that normally break down these fats. There are several types, varying in severity and age of onset, with some affecting infants and others developing later in life. Symptoms can include enlarged organs, neurological problems, and difficulty with movement or coordination. The condition is inherited, meaning it runs in families, and currently has no cure, but treatments can help manage symptoms.