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AES 2023 | The evolving landscape of rare disease research

Henry Lee, PhD, Boston Children’s Hospital, Harvard Medical School, Boston, MA, explores the evolving landscape of rare disease research. Historically, the challenges of limited knowledge, funding, and small patient populations in rare diseases have been significant obstacles, however patient advocacy groups are now driving pioneering research. Driven by the mission to make a real-world impact, Dr Lee addresses the challenges of prioritizing resources and streamlining processes in gene therapy translation. Developing transferable ideas that extend beyond specific disorders is important. For instance, the knowledge acquired through studies with succinic semialdehyde dehydrogenase deficiency (SSADHD) mouse models in Dr. Lee’s lab holds the potential to expedite treatments for related disorders like SLC6A1. Therefore, the insights gained become a valuable foundation for advancing therapeutic approaches across a broader spectrum of disorders with shared characteristics. This interview took place during the American Epilepsy Society (AES) 2023 Annual Meeting congress in Orlando, FL.

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HHC Lee is Co-Founder, Vice President, and has equity in Galibra Neuroscience, Inc. He is a co-inventor of the patent ‘Gene therapy in succinic semialdehyde dehydrogenase deficiency (SSADHD)’.