Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

In 1986, researchers discovered the gene that, when defective or flawed, causes Duchenne muscular dystrophy ... These may include: Muscle biopsy. A small piece of muscle tissue is removed and ...

Dystrophin is part of a protein complex linking the cytoskeleton of muscle fibers to the surrounding connective tissue (basal lamina ... muscle fibers are easily damaged. Duchenne muscular dystrophy ...

A new investigation into Duchenne muscular dystrophy (DMD) pathogenesis suggests that at least part of the muscle degeneration observed in DMD patients may result from the reduced production of ...

Potent in vivo gene editing in skeletal muscle of non-human primates by a novel, ultracompact CRISPR system delivered via a single AAV vector.

This damage is compounded by increased intracellular calcium levels, which impair mitochondrial function and activate calcium-dependent enzymes that degrade muscle tissue. Over time, this cascade ...

Personalized organoids — ‘mini-organs’ — may accelerate treatment for patients with rare genetic disorders. Courtesy of Europeana via Unsplash Across the globe, countless babies ...

DMD is caused by genetic mutations that prevent patients from making the structural protein dystrophin, which leads to progressive muscle wasting and is ultimately fatal. Summit is pioneering ...