A new treatment called Miplyfa offers hope for children with rare genetic disease NPC, helping stabilize symptoms and improve ...

Neimann-Pick Type C, or NPC, is a progressive genetic disorder that impacts both the body and the brain, often robbing ...

Niemann-Pick Type C, or NPC, is a relentlessly progressive and fatal genetic disorder that impacts both the body and the ...

Aqneursa has been approved to treat neurological symptoms associated with NPC in children and adults ... services manager at the National Niemann-Pick Disease Foundation. "We celebrate this ...

Zevra Therapeutics' Miplyffa has become the first medicine to be approved by the FDA for the ultra-rare genetic disorder Niemann-Pick disease ... in adults and children two years of age and ...

including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). The tech behind nizubaglustat comes from Leiden University and Amsterdam UMC in the Netherlands and led to BioGeneration ...

The fatal disease is caused by abnormal processing in body tissues of fatty substances (lipids), particularly cholesterol. Niemann Pick is also known as Children’s Alzheimer's and currently has ...

Zevra Therapeutics publishes key findings on arimoclomol's mechanism for treating Niemann-Pick disease type C in a scientific journal. Zevra Therapeutics, Inc. announced the publication of a study ...

Alford was diagnosed with Niemann Pick Type-C, an inherited disease that causes damage to the nervous system over time. According to a new release, the fatal disease is also known as Children's ...

Niemann-Pick Type C ... are living with NPC. The disease often robs patients of their ability to speak, think clearly, swallow, walk, and move. Many are children. Now there’s a new FDA-approved ...

Agents that prevent glycolipid biosynthesis have been shown to be beneficial in individuals with lysosomal storage diseases such as Niemann–Pick type C disease (NPC). In a recent study ...