HANDS (Helping Advance Neurodegenerative Disease Science)

HANDS is a consortium of patient groups, scientists, and clinicians that work together to drive the science for Sanfilippo Type C.

Patient Groups: Jonah’s Just Begun-Foundation to Cure Sanfilippo Inc., JLK-Sanfilippo Research Foundation, Sanfilippo Sud and Sanfilippo Barcelona. These are the driving forces behind the funding of the science for Sanfilippo Syndrome Type C.

Scientists: Global leaders in the research and treatment of neurodegenerative lysosomal storage disorders like Sanfilippo, HANDS’ scientists focus their resources on creating therapies and finding a cure for Sanfilippo Type C. HANDS Primary Investigators are: Alexey Pshezhetsky, Department of Pediatrics, University of Montreal; Brian Bigger, Professor of Cell and Gene Therapy at University of Manchester; and Alessandro Fraldi, Assistant Investigator at TIGEM.

The HANDS consortium relies on the advice of its esteemed team of clinicians, investigators and scientists, all of whom support, advocate and provide care for our children. Special thanks to: Sandra Alves, PhD; Wendy Chung, PhD; Graciana Diez Roux, PhD; Patricia Dickson, PhD; Sean Ekins, PhD; Maria Escolar, PhD; Paul Levy, PhD; Greg Pastores, PhD; Elsa Shapiro, PhD; Steve Wakley, PhD.

Everyone in the consortium plays an important role in creating treatments for ultra-rare diseases. The patient groups fund the science. The scientists conduct the research. The clinicians provide advice and support to the patient groups. Together, these are the people that will find a treatment and a cure. Together, these are helping HANDS.