In North Carolina, 2 years of newborn screening began with CK-MM, followed by total creatine kinase and next generation sequencing of an 86-neuromuscular gene panel, including the DMD gene ...

Duchenne muscular dystrophy (DMD) is the most common type. It’s caused by flaws in the gene that controls how the body keeps muscles healthy. The disease almost always affects boys, and symptoms ...

An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin, a protein that maintains ...

Repurposing existing therapies may help slow muscle weakness in patients with Duchenne muscular dystrophy, a new study ...

DMD has over 2.2 million bases. Gene editing is a technology that can change DNA sequences at one or more points in the strand. Scientists can remove or change a single base or insert a new gene ...

Interim results from a small group of children in a Phase I/II trial are essentially in line with that of Elevidys, according ...

RGX-202, aims to address the root cause of DMD by delivering a functional copy of the gene that encodes microdystrophin, a protein that is missing or defective in DMD patients. DMD is a form of ...

Each genetic sequence contains part of the final gene ... Meanwhile, Pfizer ditched its DMD gene therapy candidate in July after it, too, failed a phase 3 trial, which led to multiple rounds ...

The aim is to change the targeted DNA sequence to restore dystrophin protein ... delivery systems for in vivo gene editing in DMD,” the company added. Still, the slow pace of the DMD work ...