Imagine watching your child lose the ability to talk, walk, or even eat—knowing there’s a treatment out there, but the FDA keeps saying no. That’s the reality for families fighting rare diseases like Sanfilippo syndrome. Despite promising results, the FDA’s slow approvals mean kids are running out of time. My daughter finally got an experimental therapy after years of waiting, but what if she’d gotten it sooner? Families, doctors, and advocates are begging the FDA to speed things up—because every day matters. #Health #BodyHealth #RareDisease