Exploring the Potential of Gene Therapy: Is the Treatment of Rare Brain Diseases the Next Commercial Frontier?
Moving from the laboratory to the clinic, numerous treatments for uncommon neurological disorders represent the most significant trend in the development of gene therapy. Gene therapy, once a realm of scientific curiosity, has become a beacon of hope for those grappling with rare neurological diseases 1. The journey commenced over 40 years ago2, and the FDA’s …









