5 Most Promising Companies offering hope to patients with Niemann-Pick Type C
Por um escritor misterioso
Last updated 27 março 2025

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.

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

Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts

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

Orphazyme sells entire business to US biotech company — MedWatch

Researchers Study Ebola Link to Gene in Rare Disease - WSJ

Parents of twins with Niemann-Pick find a treatment

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

And Life Goes OnAn Angel and a Rainbow: October 2017

Niemann Pick Disease News, Research

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

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

What You See and What You Don't: Diagnosing Niemann Pick, Type C - Complex Child

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

Therapeutic intervention for Niemann–Pick type C disease must target
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