2024 CBER Patient and Care Partner Listening Meetings September 20, 2024

Gene therapy has recently emerged as a treatment option for some rare diseases with unmet needs. The FDA approved the first gene therapy in 2017 and 19 gene therapies as of June 2024, many of which are for rare diseases. Clinical trials that support approval of gene therapy products provide the needed information about the risks and benefits of the product so that patients and care partners can make informed decisions regarding treatment. https://www.fda.gov/news-events/2024-cber-patient-and-care-partner-listening-meetings?utm_medium=email&utm_source=govdelivery