News

UCLA researchers have developed a new method to efficiently produce and transplant functional skeletal muscle cells from human pluripotent stem cells (hPSCs). This may offer new therapeutic opportunities for patients who have muscle diseases such as Duchenne muscular dystrophy (DMD). The findings were reported in an article titled “…

Santhera Pharmaceuticals has started a campaign to help Duchenne muscular dystrophy patients  and their families learn more about the breathing challenges the condition can pose. The company’s Take a Breath DMD campaign is focusing on the importance of providing patients with proper respiratory care. It began in the United States but will…

A new way of transferring the dystrophin gene — the largest known human gene — into muscle stem cells may be possible using the human artificial chromosome (HAC) vector, a study reports, suggesting it as a potential approach for gene therapy in Duchenne muscular dystrophy (DMD). The study, “Reversible immortalisation…

Aberrant signaling in the IL-6 pathway, which is involved in muscle cell maturation, contributes to the severe symptoms seen in congenital myotonic dystrophy (CDM) patients, researchers in Japan report. The study, “Aberrant Myokine Signaling in Congenital Myotonic Dystrophy” published in the journal Cell Reports, also suggests that treatments aimed at regulating…

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…