STAT+: In the case of a devastating disease, the FDA weighs an experimental drug’s muddled data and a desperate need

Emma Albee knows the experimental drug she takes is not a cure. It won’t allow her to stop using the wheelchair she’s relied on since adolescence, and it’s not going to take away the latticework of bone that has locked her hips in place.

But for Albee and the roughly 1,000 people with a rare genetic disorder that causes their body to grow rigid bone where it doesn’t belong, the drug symbolizes a future in which years of research, advocacy, and tireless fundraising might bring about a multitude of medicines for what is now an untreatable disease.

“Even if it’s not the perfect drug, to have something where there’s data to show that it helps, that’s just so important,” said Albee, 34.

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