Joe Pierce,  —

Rare Disease Day 2023

Recent Posts

• Leaning on advocacy and friends during a difficult time of the year
• Vision problems may be earliest symptom of Sanfilippo C: Study
• Enzyme replacement therapy found effective in Sanfilippo type A mice
• My cancer is stable, so why do I feel guilty?
• OTL-201 gene therapy leads to cognitive gains in Phase 1/2 trial
• My least favorite game as a Sanfilippo mom: Playing medical detective
• Metabolites in patients’ urine may be markers of disease, ERT response
• Guest Voice: Finding gratitude while my son fights Sanfilippo type C
• Observational trial recruiting people ages 5 and up with Sanfilippo
• What if more people knew about Sanfilippo syndrome?