Study results from Sobi's previously-terminated Sanfilippo Type A enzyme replacement study have been released. The study results were published in the August 2022 edition of Molecular Genetics and Metabolism and provide some important insights. For context, this...
Replay of ADVANCE 2022, Sanfilippo conference, available on-demand
July 18, 2022
The virtual ADVANCE 2022, Sanfilippo Community Conference, July 7-8, 2022, brought together families and caregivers, scientists and researchers, clinicians and therapists, advocates, biotechs, and supporters. All to engage and advance the work to help children with Sanfilippo Syndrome. Hosted by Cure Sanfilippo Foundation, ADVANCE featured high-level collaboration, information sharing, and Sanfilippo-specific learning sessions. Topics included the role of inflammation and the immune system in Sanfilippo, new treatment approaches, Sanfilippo subtype-specific sessions, ABA and Sanfilippo, and much more. More than 450 people globally — families, scientists, clinicians, therapists, educators, pharmaceutical industry, and more — registered for ADVANCE 2022 to hear the more than 30 presentations from more than 60 speakers/panelists during the Sanfilippo conference. ADVANCE 2022 recordings of the two days of sessions are available at the link below. Each video is accompanied with a description detailing the sessions, the speakers, and the recording times for each session.
Cure Sanfilippo Foundation is grateful to the experts (scientists, clinicians, parents, and more) who shared their knowledge on a wide range of topics relevant to the Sanfilippo syndrome community. Cure Sanfilippo Foundation is also thankful for the partner sponsors that supported ADVANCE 2022: Denali Therapeutics, JCR Pharmaceutical Co., Lysogene, Orchard Therapeutics, and Ultragenyx Pharmaceutical Inc. Please stay tuned for announcements about future ADVANCE conferences. If you would like to be included in email updates from the Foundation, please let us know at email@example.com. We also welcome your input about how we can make future events, such as the Sanfilippo conference, as relevant and useful to you as possible.