NINDS Aims to Address URGenT Need for Gene Therapies

Recent successes of therapies aimed at replacing, blocking, or changing the reading of genes—referred to as gene therapy—have made real the prospect of improving outcomes for people with rare diseases.  In September 2019, NINDS held a workshop entitled “Next Generation Strategies for Gene-Targeted Therapies of Central Nervous System Disorders” to brainstorm with scientists, pharmaceutical manufacturers, and patient advocates about the best ways of getting these cutting-edge treatments into the hands of doctors and scientists to help improve the lives of patients living with rare diseases.

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