These years of work have taught us that rare disease research and clinical application in particular are expensive tasks. To think that a single organization can do it alone is unrealistic, so we decided to join parents all over the world.
Ben’s Dream , The Children’s Medical Research Foundation , The Sanfilippo Research Foundation , Team Sanfilippo , Sanfilippo Foundation Switzerland and Stop Sanfilippo have helped make it possible for the Nationwide Children’s Hospital in Ohio to reach a breakthrough in the development of gene therapy to the point of introducing a new drug to the FDA and requesting human clinical trial, scheduled for late 2014.
Alongside these foundations we have demonstrated the value and power that resides in working together for one common goal.
We face 2014 with the objective of getting a gene therapy clinical trial in Spain, developed by Hand Hayan Dr. Fu and Dr. Douglas from the Nationwide Children McArthy center of Ohio, USA.
This research group has managed to treat Sanfilippo disease in mice with very promising results, and for this reason we need to pass it to the next step, clinical testing in patients.