Researchers advance individualized treatments for neurodevelopmental diseases

Researchers are advancing toward disease-modifying treatments for neurodevelopmental disorders, specifically focusing on Angelman syndrome and the application of individualized antisense oligonucleotides (ASOs).

In a consensus statement, experts discuss the potential for 'n-of-1' ASO treatments, which are individualized genetic interventions designed to address the specific disease cause in a single patient. While these treatments are currently used in several neurodegenerative disease cases, their application in neurodevelopmental disorders raises critical questions regarding treatment eligibility, the appropriate therapeutic time window, and whether the potential benefits justify the associated risks and burdens.

Recent studies, including work from the University of North Carolina at Chapel Hill and Washington University School of Medicine in St. Louis, highlight the progress being made toward crossing the finish line for effective Angelman syndrome treatments.