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October 5, 2023 News by Lindsey Shapiro, PhD

Molecule from marine bacteria shows benefits in Sanfilippo type A

A carbohydrate molecule derived from marine bacteria was able to reduce inflammation in the brain, reverse metabolic dysfunction, and preserve brain tissue integrity in a mouse model of Sanfilippo syndrome type A, according to a new study. These benefits corresponded to an easing of motor deficits in the mouse…

January 9, 2020 News by Marta Figueiredo, PhD

Two New NAGLU-deficient Stem Cell Lines May Help Study Sanfilippo Type B

Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of Sanfilippo syndrome type B — using CRISPR-Cas9 gene editing technology. Since iPSCs have the potential to generate virtually any other cell, including nerve cells, they can be used…

Recent Posts

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  • Forever a toddler: Grieving the independent child I’d dreamed of
  • Honoring — and missing — my mother 5 years after her passing
  • FDA grants priority review to gene therapy for Sanfilippo type A
  • My daughter’s diagnosis with Sanfilippo syndrome type B
  • My sister with Sanfilippo is stable for now, so I’m taking a break
  • Feeling disconnected from my daughter as Sanfilippo progresses


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