Exondys 51 Can Preserve Pulmonary Function in Duchenne Patients, Analysis Shows

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…

Well, everyone, it is officially 2018! A new year means new beginnings, new opportunities and more chances to live the life we want. I will be honest and say that, for me, 2017 had ups and downs. There were struggles and hardships that forced me to learn some…

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…

With the holiday season in full swing, it’s the perfect opportunity to take a look back at 2017. It’s been a year full of ups and downs, and while there were definitely quite a few low moments, there were certainly quite a few highs. To celebrate the year that was,…

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…