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.

Niemann-Pick Disease Type C (NP-C) Gene Therapy Activation Meeting (GAM) - International Niemann-Pick Disease Alliance (INPDA)

Researchers Study Ebola Link to Gene in Rare Disease - WSJ

Scenic Biotech Enters Cooperative Research & Development Agreement with the National Institutes of Health for Genetic Modifier-Based Treatment Approach for Niemann-Pick Type C Disease

Biomolecules, Free Full-Text

Niemann Pick C Awareness Group

A hopeful therapy for Niemann-Pick C diseases - The Lancet

Parents of kids with Niemann Pick C advocate for adrabetadex

Autumn 2009 - the Niemann-Pick Disease Group (UK)

Gene therapy shows promise for treating Niemann-Pick disease type C1

Neurological manifestations of lysosomal disorders and emerging therapies targeting the CNS - The Lancet Child & Adolescent Health

This Post is for Laurie

Cellular Penetration and Intracellular Dynamics of Perfluorocarbon-Conjugated DNA/RNA as a Potential Means of Conditional Nucleic Acid Delivery

Niemann-Pick Canada (@NP_Canada) / X

Health Tip on New Treatment that Shows Promise in Niemann-Pick Type C Disorder - Health Tips