In DMD, the absence of dystrophin disrupts the ... function and activate calcium-dependent enzymes that degrade muscle tissue. Over time, this cascade leads to chronic muscle loss and impaired ...

DMD also can damage the heart, lungs, and other parts of the body. As they get older, your child might have other symptoms, including: The muscle problems can cause cramps at times, but in ...

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

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 ...

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

A pilot study in 19 LGMD patients and one with Becker MD found low-dose prednisone helped lower markers of muscle damage and ...

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

allowing muscle stem cells to develop into functional muscle tissue. “What makes K884 particularly promising is its precision. It targets DMD-affected cells without affecting healthy muscle stem cells ...

A live webcast will be available in the Investors & Media section of Dyne’s website at https://investors.dyne-tx.com/news-and-events/events-and-presentations and a replay will be accessible for 90 ...

Duchenne muscular dystrophy occurs when that gene fails ... These may include: Muscle biopsy. A small piece of muscle tissue is removed and examined to confirm the diagnosis or rule out another ...