Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...

Charley Seckler has found "humor in the madness" of rolling through life with a rare disease. He drops a blog post weekly, ...

Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...

The virus isn't harmful, but the gene it's carrying is known to put the brakes on the progression of DMD. The hope is that this therapy slows down Colton Belluzzo's disease, buying time for him to ...

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

But the brain is also deeply affected by DMD. Understanding these changes is crucial to developing treatments that address the full scope of the disease—not just its muscle symptoms." ...

The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.

Dietary health and weight management are important in people with DMD, given the effects the disease has on the muscles and bones, which can be worsened by the person not getting adequate nutrition.

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients ...

Trial achieved key objectives for safety, tolerability, pharmacokinetics and pharmacodynamicsClinical data from this trial, together with ...

The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...