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.
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The Company is well positioned to execute on its strategy to become a leader in cell therapy and gene therapy for rare diseases
Abeona's preclinical studies have demonstrated promising efficacy in … Learn More
September 15 - 30, 2015
CANS ACROSS AMERICA FOR SANFILIPPO SYNDROME!
If you'd like an easy to do fundraiser to help provide funding for the approved gene therapy drug to be made for our … Learn More
Our Scientific Research Advisory Board was established to provide Team Sanfilippo scientific advice, guidance and constructive criticism in our exploration of innovative research projects to fund. Learn More
Board of Directors
Our volunteer Board of Directors are mostly parents of children with Sanfilippo Syndrome who organized with one goal in mind, “to provide a better life and ultimately a cure for Sanfilippo Syndrome.” Learn More