Families racing to save their kids' lives hopeful about new FDA drug approval

After a string of denials for rare disease treatments, the FDA greenlit a drug for Hunter syndrome, a progressive illness affecting about 500 Americans.

Ever since her 15-year-old son Cole was diagnosed with a rare, progressive illness called Hunter syndrome when he was 2, Kim Stephens has been waiting. Waiting to see if Cole will lose his ability to walk. Waiting to see if Cole, who stopped speaking at about 9 years old, will lose more cognitive skills. And waiting in agony each year to see if Cole will be able to celebrate another birthday, since Hunter syndrome often kills before age 21.

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Stephens has been waiting for something else, too: hope for a medical breakthrough.

Last week, she got that when the Food and Drug Administration granted accelerated approval for a revolutionary new drug for Hunter syndrome. It was a development Stephens worried she would not see during Cole’s lifetime.