Skip to Main Content

In many ways, Emerson College freshman Meghan Waldron seems like a lot of students in Boston. She adores pop star Ed Sheeran. She loved the latest film version of “Little Women” and wants to see it 10 more times. She had a great time backpacking alone in Europe over the summer but conceded her parents were “probably freaking out.”

She also has progeria, one of the world’s rarest diseases. The fatal genetic disorder causes premature aging and has been identified in only 169 children and young adults alive today worldwide, although researchers estimate that as many as 400 have it. Most children with progeria die of hardening of the arteries, a common killer of the elderly, at an average age of just 14.

advertisement

Waldron has already lived considerably longer âۥ she turns 19 on March 1. She credits lonafarnib, an experimental medication she’s taken since 2007 in clinical trials at Boston Children’s Hospital. Eiger BioPharmaceuticals of Palo Alto, Calif., plans to complete its application for approval by March 31, with the hope of a favorable ruling from the Food and Drug Administration by year’s end. It would be the first approved drug for the ultra-rare disease.

STAT+ Exclusive Story

STAT+

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

Monthly

$39

Totals $468 per year

$39/month Get Started

Totals $468 per year

Starter

$30

for 3 months, then $399/year

$30 for 3 months Get Started

Then $399/year

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

To read the rest of this story subscribe to STAT+.

Subscribe

To submit a correction request, please visit our Contact Us page.