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Recent Posts

• It’s hard to stay positive when Sanfilippo is so cruel
• Heart condition SSS found in Sanfilippo patient for first time
• Outside, the world is pressing forward; inside, progress is still
• FDA requests manufacturing changes for UX111 gene therapy
• How my daughter with Sanfilippo handles birthday parties
• Seeing death and suffering makes me fear for my child’s future
• Sanfilippo B therapy JR-446 gets orphan drug status in Europe
• I hadn’t anticipated being a stay-at-home mom, but it works for us
• Sanfilippo B enzyme replacement therapy wins orphan drug status
• Trying to meet my son’s needs so he doesn’t feel like a ‘glass child’