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.

Push to spur drugs for deadly rare diseases

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

Parents of kids with Niemann Pick C advocate for adrabetadex

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

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

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

A rare disease patient/caregiver perspective on fair pricing and access to gene-based therapies

Family Support & Medical Conferences – NNPDF

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

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

Multiple Sclerosis Market Report 2023-2033

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

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

Orphazyme sells entire business to US biotech company — MedWatch