I am now at peace with my daughter Liv’s terminal, neurodegenerative, genetic disorder. Over four years ago, at age 2, she was diagnosed with Sanfilippo syndrome. Liv was still gaining skills at the time, but we were told that soon, it would shift, and she would start regressing and…
Regulatory authorities in the U.K. have granted innovation passport designation to OTL-201, an experimental gene therapy for Sanfilippo syndrome type A, generally the disease’s most severe form. This new status gives treatment developer Orchard Therapeutics (recently acquired by Kyowa Kirin) access to a U.K. program…
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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