Gemma Biotherapeutics ("GEMMABio"), a clinical‑stage, global, genetic medicines company, today announced the presentation of preclinical data supporting candidate declaration for GB703, a novel, ...

Scientists have created a new gene therapy for Duchenne muscular dystrophy (DMD) that may not only help stop the disease in DMD patients, but might also help restore their damaged muscles in the ...

Regenxbio’s gene therapy for Duchenne muscular dystrophy has smashed the primary endpoint of its pivotal trial, securing a ...

Regenxbio is pitching at FDA approval next year for its Duchenne muscular dystrophy (DMD) gene therapy RGX-202, after ...

There is a new game changer for children with a certain form of muscular dystrophy. Now, a child who was destined to end up ...

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

Muscular dystrophy is a disease caused by mutations in the dystrophin gene. The muscles of muscular dystrophy patients weaken and atrophy over time. Heart and breathing muscles may also eventually be ...

Editor’s note: This is an automatically generated transcript. Please notify editor@healio.com if there are concerns regarding accuracy of the transcription. We still have maybe 15% or so of people who ...

Myotonic dystrophy is thought to be caused by the binding of a protein called Mbnl1 to abnormal RNA repeats. In these two images of the same muscle precursor cell, the top image shows the location of ...

Muscular dystrophy (MD) is a group of genetic diseases that cause your muscles to progressively weaken and degenerate. There are several types of MD, each with its own symptoms, but they all involve ...