Tests of NT-3 gene therapy on mouse models suggest potential to treat a host of muscle-wasting conditions.
A new study shows that cannabidiol is effective in children with Lennox-Gastaut syndrome.
Recent applications of adeno-associated virus (AAV) mediated gene therapy have focused mainly on correcting recessively inherited diseases. But what about dominantly inherited disorders? It looks like AAV could be a delivery mechanism for treating those genetic disorders, too.
The phase 1 clinical study shows that gene therapy extends survival of patients and supports achievement of milestones previously unseen in the natural course of the disease.
Two studies demonstrate clinically significant response to oral liquid cannabidiol over placebo.
Clinical implications include specialized testing for deficits in executive function as these may not be apparent at a routine exam.
