Rare disease trials can't find enough patients. It's forcing the FDA to rethink its approach
When Hannah Sames was 4 years old, she was diagnosed with an ultra-rare genetic condition called giant axonal neuropathy. She’s 18 now, and her doctors expected long ago that she would be quadriplegic, and consigned to an early death.
Instead, she can stand, albeit with help. “Even with her physical challenges, she’s happy. She’s a typical teenager who is social and loves music,” said her mother, Lori Sames.
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