News

Apple Tree Partners has invested $40 million in Stoke Therapeutics to come up with gene-expression treatments for muscular dystrophy and other genetic disorders. Stoke aims to develop antisense oligonucleotides to promote targeted augmentation of nuclear gene output (TANGO). The goal is to reverse the features of rare diseases…

Expansion Therapeutics has raised $55.3 million in Series A financing to advance its portfolio of drugs targeting several genetic disorders, including myotonic dystrophy type 1 (DM1), the leading cause of adult onset muscular dystrophy. Toxic levels of RNA are thought to…

Researchers have developed a new “checkpoint” model that may be used to discover potential therapies for disorders such as Duchenne muscular dystrophy (DMD) and cystic fibrosis. The research, “Aminoglycoside-mediated promotion of translation readthrough occurs through a non-stochastic mechanism that competes with translation termination,” was published in the journal Human…

Researchers tracked the way adult skeletal muscle stem cells (MuSCs) multiply in response to aging or injury. The study has implications for treatments in diseases of muscle tissues, such as muscular dystrophies. The study, “Muscle Stem Cells Exhibit Distinct Clonal Dynamics in Response to Tissue Repair and Homeostatic…

Treatment with Exondys 51 (eteplirsen) can prevent lung function decline in patients with Duchenne muscular dystrophy (DMD), according to the results of an analysis of clinical trial data. The findings were reported in a study titled “Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison…

Scientists at King’s College London, UK discovered that the PAX7 gene and its regulatory gene network is a new hallmark of facioscapulohumeral muscular dystrophy (FSHD). Moreover, PAX7 works better as a biomarker for the disease than the current leading candidate gene…

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…