Challenges of Research into CNS Manifestations of Metabolic Diseases

Jointly sponsored by the NIH’s Office of Rare Diseases Research (ORDR) and the FDA’s Center for Drug Evaluation and Research, Division of Gastroenterology and Inborn Errors of Metabolism Products (DGIP), the goal of the workshop is to bring together those involved in research and investigational treatments for central nervous system aspects of inborn errors of metabolism, and to discuss ways to translate these therapies to clinical trials and beyond.