trelarosa,  —

Recent Posts

• 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
• FDA puts Sanfilippo A enzyme replacement therapy on fast track
• Milestones can bring unexpected tides of grief with Sanfilippo
• High school was a turning point for me and my Sanfilippo sister
• Connecting with my daughter is difficult due to Sanfilippo syndrome
• FDA clears Phase 1 trial of GC1130A for Sanfilippo type A