Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.
Clinical Trials as Topic
National Institutes of Health (U.S.)
Translational Medical Research
United States Food and Drug Administration
Gene therapy has shown clinical efficacy for several rare diseases, using different approaches and vectors. The Gene Therapy for Rare Diseases workshop, sponsored by the National Institutes of Health (NIH) Office of Biotechnology Activities and Office of Rare Diseases Research, brought together investigators from different disciplines to discuss the challenges and opportunities for advancing the field including means for enhancing data sharing for preclinical and clinical studies, development and utilization of available NIH resources, and interactions with the U.S. Food and Drug Administration.