New treatment for Duchenne muscular dystrophy brings hope to a West Virginia boy

Six-year-old Alexander Shell loves tools, cars and building things. He also has Duchenne muscular dystrophy (DMD), one of the most severe forms of inherited muscular dystrophies. DMD leads to progressive muscle weakness that may advance to the point where affected patients cannot carry out the activities of daily living and must use wheelchairs; cardiac, respiratory and orthopedic complications are also common. Most patients with DMD usually don’t make it past their twenties.

But thanks to West Virginia University Medicine Children’s, there is hope for Alexander. In April he was among the first patients in the country — and the first in West Virginia — to receive the first dose of ELEVIDYS. Early studies of this prescription gene therapy treatment, which received an accelerated approval from the FDA, aim to slow the progression of DMD, as well as improve motor functions in patients. While long-term studies still need to be done, the hope is that the medication will slow progression of the disease, enabling those with DMD to live longer, healthier lives.

For more information about Alexander and his treatment, click here.