5 Most Promising Companies offering hope to patients with Niemann-Pick Type C

Niemann–Pick is a rare autosomal recessive inherited disease that is characterized by excessive accumulation of cholesterol and lipids in cells, leading to apoptosis and neural degeneration. The disease affects numerous body parts such as the brain, nerves, liver, spleen, bone marrow and lungs. Those suffering from the disease suffer from lack of coordination, dystonia; sleep disturbances, difficult in eating and swallowing and frequent bouts of pneumonia.

Valley family fights for drug to save sick child

And Life Goes OnAn Angel and a Rainbow: October 2017

PDF) Laboratory diagnosis of the Niemann-Pick type C disease: an inherited neurodegenerative disorder of cholesterol metabolism

Precision medicine in monogenic inflammatory bowel disease: proposed mIBD REPORT standards

And Life Goes OnAn Angel and a Rainbow: October 2017

Two-Year-Old Kamryn Living with Niemann-Pick Disease Type C (NPC) - The Children's Inn at NIH

Investigational New Drug (IND) - The Addi and Cassi Fund

40th Anniversary Report by metabolicsupportuk - Issuu

Parents of kids with Niemann Pick C advocate for adrabetadex