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.

Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann–Pick Type C Using Quantitative Proteomics

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

40th Anniversary Report by metabolicsupportuk - Issuu

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

Advancements in Niemann-Pick Disease Type C Treatments

A terrible disease, children at risk, and a promising treatment that's about to vanish: Part II

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

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

2022 drug approvals: Biopharma delivered 34 new drugs

Researchers Study Ebola Link to Gene in Rare Disease - WSJ

Current Clinical Applications of In Vivo Gene Therapy with AAVs - ScienceDirect

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

Pharmaceuticals, Free Full-Text

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