Miglustat may improve swallowing ability in NPC1 patients

Miglustat may improve swallowing ability in NPC1 patients

Miglustat may stabilise the problems of swallowing ability that occur in children and adolescents with Niemann-Pick type C1 (NPC1), a rare and ultimately fatal neurological disease, according to a recent study published in JAMA Neurology.

The study which was conducted by researchers at the National Institutes of Health found 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.
Miglustat is indicated for the treatment of progressive neurological manifestations associated with the autosomal recessive, neurodegenerative disease in both adults and children. Previous studies have suggested that by slowing this neurological deterioration, miglustat can stabilise swallowing ability.

In the current study, researchers used video fluoroscopy — an X-ray scan of the throat — to evaluate swallowing function in 120 NPC1 patients. They ranked each of the scans based on the need for feeding tubes and other interventions to assist swallowing and on the likelihood and extent of food and drink entering the airway.

On average, patients were evaluated once a year for three years. Of these, 36 had been prescribed miglustat and 24 had not. The findings revealed that, compared to those who did not take the drug, the cohort which took the drug had a 91% lower risk for deterioration in swallowing and a 72% lower risk for getting food or drink in the airway.