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.
On May 24, 2019, Zolgensma, a first-of-its-kind gene therapy for spinal muscular atrophy was approved.
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.
Measures taken in first 12 hours of life are associated with a host of deficits.
A quality improvement project with relatively simple interventions resulted in a significant decrease in Emergency Department visits and inpatient admissions, while saving $2 million in health care costs.
