News

Scientists at Saint Louis University believe that an investigational therapy called SR8278 holds potential for treating Duchenne muscular dystrophy (DMD) after it was shown to increase muscle function and decrease muscle fibrosis in lab mice. The discovery follows the team’s research of the nuclear receptor REV-ERB, which regulates key processes in…

Researchers have found that lowering the the levels of a protein called sarcolipin lessens muscle weakness and improves skeletal and cardiac muscle function in a mouse model of Duchenne muscular dystrophy (DMD). These findings suggest that therapeutics targeting sarcolipin may have significant benefits to DMD patients. The study “Reducing sarcolipin expression…

Researchers have found that skeletal muscle magnetic resonance imaging (MRI) correlates with motor function and can help predict the degree of functional deterioration in patients with Becker muscular dystrophy (BMD). A study with that finding, “Muscle MRI and functional outcome measures in Becker muscular dystrophy,” was published in the…

Through Dec. 31, Parent Project Muscular Dystrophy (PPMD) will match all donations to expand its ongoing Gene Therapy Initiative in an effort to raise $400,000 to fund research projects seeking to advance gene therapy as a treatment for Duchenne muscular dystrophy (DMD). Gene therapy has been increasingly studied…

Researchers found that giving Duchenne muscular dystrophy (DMD) patients glucocorticoid therapy keeps them mobile for a longer period of time and reduces their risk of dying. Their study, “Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study,”…

Clay Matthews of the Green Bay Packers took to the field in Week 13 of the NFL season wearing a cause close to his heart on his feet, as he joined the football league’s “My Cause My Cleats” campaign to raise funds for Duchenne muscular dystrophy (DMD). “My…

Karen Haberberg, a New York portrait photographer, has taken tens of thousands of pictures throughout her career of families, couples, pregnant women, teenagers, and fashion models — not to mention dramatic landscape shots from Iceland to Indonesia. She’s also done several personal photo projects including “Benjamin,” a haunting portrayal…

Solid Biosciences announced the pending start of a first clinical trial to test the company’s leading investigational therapy – called SGT-001, a microdystrophin gene transfer candidate, as a potential treatment for Duchenne muscular dystrophy (DMD). The Phase 1/2 trial, called IGNITE DMD, will soon open for patient enrollment in the…

Capricor Therapeutics has received a green light from the U.S. Food and Drug Administration to launch a Phase 2 study of CAP-1002 cell therapy in children and adults with advanced Duchenne muscular dystrophy. Capricor expects to open the trial to patient enrollment in early 2018 and, if successful,…

The first healthy subject was dosed in a Phase 1b clinical trial evaluating the safety, tolerability and drug properties of ELX-02 as a candidate for the treatment of multiple conditions, including Duchenne muscular dystrophy (DMD). The multiple ascending dose (MAD) study (NCT03309605) is being led by Sevion…