July15, 2019 - Team Sanfilippo (TSF Inc ) and Seelos Therapeutics are finalizing the last few things so that the trial can begin! We are very excited to get this started to say the least This is the first trial that was/is open all types of Sanfilippo, so far only Types A and B have contacted us for enrollment This is the first trial that has shown to be safe and have efficiency in similar diseases in humans already! This is the first trial designed and funded by a single patient organization with the support of a therapeutic company! We are proud to be partners with Seelos Therapeutics Once the trial begins, within approx 3 months of that time, we are expecting to ...Read More
What is Sanfilippo Syndrome?
Sanfilippo syndrome is a genetic error of metabolism. It is a mucopolysaccharidoses, an MPS disorder, MPSlll. Mucopolysaccharides are long chains of sugar molecules, which are used in building connective tissues. When the body is finished using these molecules, it breaks them down, with enzymes, and disposes of them. Children with Sanfilippo syndrome are missing or are deficient in the enzyme to break down the molecules. Instead, the body stores these molecules in the cells. This storage causes progressive damage.