Micro-dystrophin gene therapy improves survival but may pose long-term risks
Micro-dystrophin gene therapy greatly extended lifespan and improved heart function in severe DMD preclinical models.
Micro-dystrophin gene therapy greatly extended lifespan and improved heart function in severe DMD preclinical models.
PBGENE-DMD could help up to 60% of patients with Duchenne muscular dystrophy by permanently editing their dystrophin gene.
A new gene therapy using triple AAV vectors restored full-length dystrophin and improved muscle function in mice with DMD.
Stress during late pregnancy changed how Duchenne muscular dystrophy (DMD) impacted offspring in a mouse model.
Elevidys is a one-time gene therapy that helps patients with DMD produce a key muscle protein and is under ongoing safety evaluation.
CRISPR shows promise in correcting DMD mutations and restoring dystrophin, and future patients may benefit from personalized strategies.
For people with DMD, physical activity is about more than walking, with independence and quality of life tied to meaningful movement.
Muscle weakness in Duchenne muscular dystrophy (DMD) does not follow a simple decline, but has periods of stability before worsening.
Researchers identified two previously unreported mutations in the Dystrophin gene, providing new insights into muscular dystrophy.
Patients of different racial and ethnic backgrounds with DMD received different treatments and had varying health care costs.