Cure Sanfilippo Foundation的动态

Don’t miss the packed agenda of ADVANCE 2024, the virtual Sanfilippo community conference, Oct. 29 & 30, hosted by Cure Sanfilippo Foundation. Register today! Don’t miss hearing from and interacting directly with experts who are leading research around the globe, clinicians pioneering best-in-class therapeutic care, and the families of children with Sanfilippo Syndrome. When: Oct. 29 & 30, 2024 Where: Virtual, so wherever you are Cost: Free Register: www.ADVANCESanfilippo.com This year’s packed agenda includes sessions on: - The Eyes Have It: Impact on the retina and monitoring strategies in Sanfilippo disease - Affected individuals and families share their personal viewpoints and experiences - Nutrition to support a healthy body and brain - Immune responses, ethics of dose escalation, and community/patient perspectives on gene therapy risk/benefit & long-term follow up - Energy metabolism and secondary mitochondrial dysfunction in Sanfilippo - Emerging model system of the peripheral nervous system - What does “Accelerated Approval” mean? - Updates on drug-repurposing and enzyme-restorative clinical trials See the full agenda for this two-day, Sanfilippo-specific conference and register today at www.ADVANCESanfilippo.com. As always, there is no cost to attend ADVANCE. Cure Sanfilippo Foundation believes in making this wealth of Sanfilippo-specific information available to all. Many thanks to our sponsors, Denali Therapeutics, JCR Pharmaceuticals, GC Biopharma (GC???), @Novel Pharma, Orchard Therapeutics - U.S., and Ultragenyx, for assisting with the event expenses so it can be freely available to all. New this year: real-time translations will be available in Spanish, during the live conference! Recordings of ADVANCE 2024 sessions will be made available after the conference and have an option of translated captions in a wide range of languages. Make sure to register, even if you can’t attend live, so you receive the link to the recordings right away. www.ADVANCESanfilippo.com

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