Researchers at Oregon State University and other institutions have discovered a type of drug delivery system that may offer new hope for patients with Niemann Pick Type C1 disease.
No treatment currently exists for the disease, however, a compound that shows promise is now undergoing clinical trials, but it has major drawbacks.
New findings, published today in Scientific Reports, outline the potential for a nanotech-based delivery system to carry the new drug into cells far more effectively, improve its efficacy by about five times, and allow use of much lower doses that may still help treat this condition without causing such severe hearing loss.