DMD occurs because the body is unable to make dystrophin, a protein necessary for maintaining muscle cells and keeping them ...

Dysfunctional dystrophin, the muscle protein affected in Duchenne, leads to an important choice for patients about how to ...

Muscle biopsy. Using a needle, the doctor removes a tiny piece of your child’s muscle. They’ll look at it under a microscope to check for low levels of dystrophin, the protein that is missing ...

Without dystrophin, your muscle cells remain unprotected and are at risk of damage. Becker muscular dystrophy is a genetic disorder linked to the X chromosome. The dystrophin gene that causes it ...

Muscle biopsies from 8 patients, all of whom were ambulatory and had biopsies at 24 and 48 weeks, showed stabilized dystrophin expression between 24 and 48 weeks of dosing. The mean was 7.8% (95% ...

The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...

Aug. 24, 2023 — Duchenne muscular dystrophy (DMD) is a muscle degeneration disorder caused by mutations affecting the dystrophin gene. Researchers show how a dual CRISPR RNA method restored ...

"Our data confirmed we successfully restored full-length dystrophin in both the skeletal and heart muscles of mice with DMD, leading to significant improvements in their muscle health, strength ...

“Cardiac muscle expression is a critical consideration, as it has been shown that restoration of dystrophin in skeletal muscles but not cardiac muscle will exacerbate cardiomyopathy, sometimes ...

In DMD, low levels of dystrophin protein cause the heart to grow bigger and the heart muscle to weaken. These changes make it harder for the heart to pump blood.

If needed, a muscle biopsy (removal of a small muscle sample) can be performed, to show the absence of dystrophin in the muscles. However, muscle biopsies are less common today since most diagnoses ...