What about Duchenne's girls?

Duchenne muscular dystrophy is a disease associated with young boys and young men: The degenerative disorder, whose patients rarely live past 30, seldom manifests in females. So the clinical trials tend to focus only on males, and fundraisers are often gender-specific: “Run for Our Sons” is one of the more prominent events to raise awareness of Duchenne.

But Deb Jenssen, a mom of triplets in Madison, Ala., feels that the medical community may be excluding her daughters. Two of her three children carry the genetic mutation that leads to Duchenne, and are showing symptoms of the disease — but, given their gender, they have not been considered patients with Duchenne muscular dystrophy. Jenssen has been attempting to enroll her children in clinical trials and has come up short.

So, in an effort to curb the degenerative symptoms of her daughters’ disease, Jenssen is speaking out to Duchenne drugmakers and medical researchers:

“If nobody knows about us,” Deb Jenssen told STAT’s Andrew Joseph,” nobody’s going to help us.”