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.

Therapeutic intervention for Niemann–Pick type C disease must target

Topic · Rare disease awareness ·

Presentations of Niemann-Pick Type C disease

Frontiers Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies

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

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

Advancements in Niemann-Pick Disease Type C Treatments

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

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

Endpoint Considerations to FacilitateDrug Development for Niemann-Pick Type C (NPC)

Researchers Study Ebola Link to Gene in Rare Disease - WSJ

PDF) Synthetic high-density lipoprotein nanoparticles for the treatment of Niemann–Pick diseases

Niemann Pick Disease News, Research

40th Anniversary Report by metabolicsupportuk - Issuu