Inside STAT: They rallied around ‘our boys’ with Duchenne. Where did that leave girls? 

THE JENSSEN FAMILY AT A UNIVERSITY OF ALABAMA FOOTBALL GAME IN TUSCALOOSA. (COURTESY JENSSEN FAMILY)

Duchenne muscular dystrophy has been thought of as a boys’ disease. After all, 1 in 5,000 boys are at risk of developing the debilitating genetic disorder, but among the female carriers, fewer than 5% lose their ability to walk. It’s perhaps no surprise, then, that all treatments and trials have focused on helping boys. But where does that leave the few girls who do show symptoms of the disease? That’s the question that’s been plaguing the Jenssen family: Two of their triplet girls are showing signs of the disease. So when a new drug trial promised to be inclusive of all kinds of patients with Duchenne, the Jenssens were excited. Until they were told that all patients meant likely meant all boy patients. STAT’s Andrew Joseph has the story from Madison, Ala.
“Simply the threat of a revised public charge sent fear throughout the immigrant community and had a chilling effect on health care utilization,” says Dr. Sarah Polk of Johns Hopkins University.