After 20 years since finishing undergraduate studies, Carol Wilkinson, MD, PhD, IDDRC Investigator, has observed that the therapies for autistic children with limited communication are, unfortunately, approximately the same today. In part, this is due to the derth of quality research data needed to inform the development of new treatments. What are the barriers to gathering quality data? Dr. Wilkinson explores this question and possible answers.

Important insights into Hereditary Spastic Paraplegia and key findings that may lead to novel therapies.

New Gene Discovery for syndromic intellectual disability has been described. Researchers at the RSZ-Translational Neuroscience Center @BostonChildrens are leading efforts to better understand this disorder, recently named “Chopra-Amiel-Gordon syndrome.”

Dr. Lipton's reserach centers on the molecular and cellular interfaces between circadian rhythms and neurodevelopmental and neurodegenerative disorders

Promising Gene Therapy Pathways for Combating Central Nervous System Diseases. Dr. Zhigang He, Professor of Neurology and Ophthalmology, Harvard Medical School, discusses how developing efficient BBB-penetrating recombinant AAV vectors shows great promise for enabling gene therapy for treating Central Nervous System (CNS) diseases including those relating to intellectual and developmental disabilities.

The Rosamund Stone Zander Translational Neuroscience Center at Boston Children's Hospital will transform the field as the first of its kind pediatric-focused translational neuroscience center.