Hannah Sames (courtesy photo)

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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