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Recent Posts
- 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’
- Balancing logic and emotion isn’t easy as a Sanfilippo parent
- Spruce to move tralesinidase alfa into Phase 3 clinical study