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.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Already have an account? Log in

Individual plans

Group plans

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $39/month

$30 for 3 months Get Started

Then $39/month

Annual

$399

Save 15%

$399/year Get Started

Save 15%

11+ Users

Custom

Savings start at 25%!

Request A Quote Request A Quote

Savings start at 25%!

2-10 Users

$300

Annually per user

$300/year Get Started

$300 Annually per user

View All Plans

Get unlimited access to award-winning journalism and exclusive events.

Subscribe

Log In