The U.S. Food and Drug Administration (FDA) approved the gene therapy Fayuvi (rebisufligene etisparvovec-hopf, formerly known as UX111) as the first treatment for Sanfilippo syndrome type A. Ultragenyx Pharmaceutical said it expects the drug to be shipped to qualified treatment centers (QTCs) — institutions with expertise and training…
Discussion
My son, Liam, has started asking more questions about why his sister behaves differently from his friends. I knew this time would come, when I’d have to start answering the “why” questions. My daughter, Liv, has Sanfilippo syndrome, a neurodegenerative genetic disorder. She is 6 years old, but her…
Discussion
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…
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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