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Recent Posts
- FDA approves new gene therapy as first treatment for Sanfilippo A
- My son is starting to ask questions about his special needs sibling
- Groups invest $5.5M to expand access to experimental Sanfilippo B drug
- Moving may be good for our daughter, but the thought of it seems daunting
- Online research platform for Sanfilippo families goes live
- Thinking about my daughter’s future includes planning her funeral
- Enzyme therapy protects kids’ brain function for up to 6 years
- Accepting my daughter’s Sanfilippo diagnosis has allowed me to heal
- Sanfilippo A gene therapy candidate granted access to UK’s ILAP program
- I’m a wife and mom to a special needs child, but what defines me?