Viltolarsen targets dystrophin gene mutations, enabling functional dystrophin protein production, crucial for muscle health in patients with DMD. Consistent viltolarsen treatment slowed disease ...
New data suggest that givinostat treatment may slow the decline of upper limb function in boys with Duchenne muscular ...
(Headquarters: Paramus, New Jersey; President: Yukiteru Sugiyama), a biopharmaceutical leader in rare disease and subsidiary of Nippon Shinyaku Co., Ltd. (Headquarters: Kyoto, Japan; President: Toru ...
Treatment with deramiocel led to a 54% slowing of upper limb function deterioration and a 91% slowing of cardiac function deterioration compared with placebo. Topline data were announced from a phase ...
Capricor Therapeutics has presented positive long-term data from its HOPE-2 clinical trial at the 2025 Muscular Dystrophy Association Conference, indicating that its leading treatment, deramiocel, can ...
Investigators evaluated longitudinal MRI and spectroscopy outcomes and ambulatory function among 180 patients with Duchenne muscular dystrophy (DMD) to establish the utility and reproducibility of ...
There are many kinds of muscular dystrophies, which are caused by genetic mutations. These diseases reduce mobility, and complicate everyday tasks. Various types of muscular dystrophies can affect ...
(RTTNews) - Capricor Therapeutics, Inc. (CAPR), on Monday, announced positive data from its ongoing HOPE-2 open-label extension trial, demonstrating the long-term efficacy of Deramiocel for the ...
Cardiomyopathy is the leading cause of mortality in patients with DMD. Compared with their healthy counterparts, patients with DMD — including those with preserved left ventricular ejection fraction ...
Researchers at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy. Duchenne muscular dystrophy (DMD) is a ...
New research recommends daily steroid doses for children with Duchenne muscular dystrophy, marking a significant change in how the disease is treated. New research published in JAMA recommends daily ...
Using a new gene-editing technique, a team of scientists from UT Southwestern Medical Center stopped progression of Duchenne muscular dystrophy (DMD) in young mice. If efficiently and safely scaled up ...
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