Cure Sanfilippo Foundation and the National MPS Society are investing $5.5 million to expand access to an experimental treatment for children with Sanfilippo syndrome type B. The funding will support a U.S.-based expanded access program expected to launch as early as October for tralesinidase alfa (TA-ERT), an…
Discussion
When we received my daughter Liv’s Sanfilippo syndrome diagnosis four years ago, my mind raced with all the changes that would come — the therapies and medical appointments — and just how different her future suddenly looked. In an instant, her diagnosis had changed our lives forever. Liv was…
Discussion
Enrollment is now open for SanfilippoLINK, a global clinical research and patient registry platform that aims to advance scientific understanding of Sanfilippo syndrome. The platform, operated by the Cure Sanfilippo Foundation, is open to people with all types of Sanfilippo syndrome. The platform consists…
Discussion
As morbid as it sounds, I sometimes find myself thinking about my daughter’s death and funeral. In 2022, Liv was diagnosed with Sanfilippo syndrome, a terminal, neurodegenerative, genetic disorder. Today, she is 6 and doing well, all things considered, but this diagnosis has felt like nothing short of a…
Sanfilippo syndrome is a genetic disorder that leads to a form of childhood dementia. Learn more about the disease here.
Sanfilippo is caused by mutations in one of four genes that produce the enzymes needed to break down heparan sulfate. Learn more about what causes the disease here.
Symptoms range from behavioral problems to developmental delays and sleep issues. Learn more about the symptoms of the disease here.
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