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 ...

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

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 ...

There is an unmet need for therapies targeting Duchenne muscular dystrophy (DMD)–related heart disease, and phase 2 results ...

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

The GoFundMe page was set up to raise money to cover the cost of life-saving treatment for the 14-year-old boys, who suffer ...

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

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 ...

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

PepGen (PEPG) “provided recent updates on its CONNECT clinical program investigating PGN-EDO51 in Duchenne muscular dystrophy for patients ...

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 ...