Ruth Doris,  —

Recent Posts

• Grappling with the fear that I’ll get blindsided once again
• Sanfilippo type A children show gains with gene therapy UX111
• As our son with Sanfilippo ages, we continue to visit the Easter Bunny
• The emotional toll of having a child with Sanfilippo syndrome
• Developing Sanfilippo, MPS disorder biomarkers aired in workshop
• How accessibility helps me make memories with my son
• Sanfilippo syndrome type A candidate named orphan drug
• Holiday travel required conquering a fear of leaving my son at home
• A new year brings uncertainty for Sanfilippo families
• JR-441 enzyme replacement therapy named orphan drug by FDA