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A Phase 1 clinical trial in Japan that’s testing JCR Pharmaceuticals‘ experimental enzyme replacement therapy JR-441 for treating Sanfilippo syndrome type A has dosed its first patient. The study will test the therapy as a once-weekly infusion into the bloodstream in children and adolescents, ages 1-18 to…
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“Are you ready?” I asked my husband as we stood on the steps outside of a massive, intimidating building. “As ready as I can be, I guess,” he replied. We nodded at each other and walked to the front door. It was time to attend the first high school…
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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