Team Sanfilippo Foundation was started by a group of parents with the mission of finding potential therapies that can be tested clinically in the near future. It is our desire to advance research, education, improve the quality of life for the children affected by this disorder and ultimately find a cure.
Due to recent scientific advancements in the areas of enzyme replacement, gene therapy, and small molecules which has led to the initiation of clinical programs for Sanfilippo Syndrome we are focused on the development of combined therapies to give children the best possible treatment strategies.
Help us realize this focus and bring the gene therapy program at Nationwide Children's Hospital to clincial trial by buying a glass of Purple Lemonade. Thank you.