A new study in the peer-reviewed journal Human Gene Therapy showed that more than half of individuals with Niemann-Pick disease, type C1 (NPC1) who were tested lacked neutralizing antibodies ...

The TransportNPC™ study is considered the most comprehensive of its kind concerning patient size, global reach, duration, and clinical outcomes for NPC1 treatment. The stock has shown resilience ...

NPC1 is a rare, fatal neurodegenerative disorder, for which systemic AAV9-based gene therapy in mouse models has shown some success. The presence of neutralizing antibodies against AAV can reduce ...

Background: Genetic studies have implicated the NPC1 gene (Niemann Pick type C1) in susceptibility to obesity. Methods: To assess the potential function of NPC1 in obesity, we determined its ...