From National Institutes of Health, September 9, 2020
The drug miglustat appears to stabilize the swallowing problems that occur in children and adolescents with Niemann-Pick type C1 (NPC1), a rare and ultimately fatal neurological disease, according to a study by researchers at the National Institutes of Health. The authors conclude that the drug could slow the deterioration of swallowing function in NPC1 cases and decrease the risk of pneumonia resulting from aspiration, or inhaling food or drink. Aspiration pneumonia accounts for roughly 2 out of 3 deaths in people with NPC1.
The NTA is working with companies, clinicians and the FDA in hopes of having Miglustat be an indication for NPC disease.