The family of Connor Dobbyn created a video time capsule to document the many efforts and many, many kind people it took for the Save Connor campaign to reach $1 million. All to help fund the first-ever gene therapy clinical trial for Sanfilippo Syndrome Type C.
Denali ERT clinical trial, Sanfilippo Type A, UCSF site activated
Cure Sanfilippo Foundation is happy to share that we've been notified by the institution that a clinical trial site for Sanfilippo Type A has opened at the University of California-San Francisco (UCSF) in the U.S. This trial is sponsored by Denali Therapeutics. It...