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Recent Posts
- First Sanfilippo syndrome type A patient dosed with JR-441 ERT
- With Sanfilippo, it’s a foreign feeling when things go right
- With Sanfilippo, we’re like a plane stuck on the tarmac
- Neurodegeneration-linked proteins accumulate across Sanfilippo types
- Good news about our son’s weight — and our caregiver burnout
- Japan agency clears JR-441 trial for Sanfilippo syndrome type A
- When Sanfilippo gets in the way of making memories with family
- Advocating for an adult with Sanfilippo is tiring, but necessary
- Denali’s DNL126 selected by FDA for START pilot program
- Hurricane Beryl reminds us of all we take for granted