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https://teamsanfilippo.org/what-is-sanfilippo-syndrome
Sanfilippo syndrome is a progressive disorder, meaning that at birth the children appear typical and do not show signs of the disorder. As the disease progresses the children degenerate, losing the ability to speak, walk, eat and eventually lose their lives.
https://www.mpstransphera.com/
If your child has been diagnosed with Sanfilippo syndrome (MPS III), they may be able to participate in a study for a one-time gene therapy. Scientists can deliver a working copy of the gene to children with Sanfilippo syndrome (MPS III) using this new approach.
https://mpssociety.org/learn/diseases/mps-iii/
MPS III is a mucopolysaccharide disease also known as Sanfilippo syndrome. It takes its name from Dr. Sylvester Sanfilippo, one of the U.S. doctors who described the condition in 1963. It takes its name from Dr. Sylvester Sanfilippo, one of the U.S. doctors who described the condition in 1963.
https://curesanfilippofoundation.org/
Cure Sanfilippo Foundation leads in driving research to accelerate discovery of a cure for Sanfilippo Syndrome. In just six years, generous donors like you have enabled Cure Sanfilippo to generate more than $8 million through grassroots and viral fundraising to support and/or architect 20+ research projects globally, including several clinical trials.
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