Rare Disease Day 2023
- 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?