Recent Posts

• HSPC transplant eases disease symptoms in Sanfilippo C mice
• Forever a toddler: Grieving the independent child I’d dreamed of
• Honoring — and missing — my mother 5 years after her passing
• FDA grants priority review to gene therapy for Sanfilippo type A
• My daughter’s diagnosis with Sanfilippo syndrome type B
• My sister with Sanfilippo is stable for now, so I’m taking a break
• Feeling disconnected from my daughter as Sanfilippo progresses
• UX111 offers cognitive, motor skill gains in Sanfilippo syndrome type A
• Ultragenyx asks FDA to review gene therapy for Sanfilippo A
• Managing volatile emotions is part of being a Sanfilippo caregiver