Tevard Biosciences aims to use transfer RNA (tRNA) to modulate RNA function to treat Dravet syndrome and other rare diseases.
Tim and Laura Revell have raised millions of dollars for CureDuchenne to research a disease that affects both their sons.
The American College of Obstetricians and Gynecologists now recommends that all women—ideally before pregnancy—be screened for hemoglobinopathies.
Edgewise Therapeutics focuses on small-molecule therapies to preserve muscle in patients with DMD, while Locanabio is working on snRNA technology.
Two boys with DMD—one in Connecticut and one in California—have shown remarkable results in the trial of a gene therapy from Pfizer.
A researcher at Baylor College of Medicine is studying whether cannabidiol (CBD) could help boys and young men who have DMD.
Julie Parsons, MD, of Children’s Hospital Colorado, expects a surge in demand if the Food and Drug Administration approves a new gene therapy for Duchenne.
Randi Clites of Little Hercules Foundation has made it her mission to get DMD newborn screening implemented in all 50 states.
The nation’s first CureDuchenne Clinic has opened in a suburb of Dallas, Texas, to treat Hispanic patients with rare neuromuscular diseases.