News

FibroGen, a biotechnology company with expertise in connective tissue growth factor (CTGF) and hypoxia-inducible factor (HIF) biology, recently announced enrolling the first two patients in its new, open-label, multicenter Phase 2 clinical trial to investigate the FG-3019 compound in Duchenne muscular dystrophy (DMD) patients.  The trial is currently recruiting participants and further…

Researchers have found that clinically significant findings of cardiac arrhythmia, as measured by Holter findings, are rare even among young Duchenne muscular dystrophy (DMD) patients with moderate cardiac dysfunction. The study, titled “Ambulatory Monitoring and Arrhythmic Outcomes in Pediatric and Adolescent Patients With Duchenne Muscular Dystrophy”, was published in the …

A drug used to treat leukemia might be a potential therapy to slow the progression of Duchenne muscular dystrophy (DMD), according to the results of a study published in the journal Human Molecular Genetics and titled “Dasatinib as a treatment for Duchenne muscular dystrophy.” In the study, Dr Leanne…

Cambridge, Massachusetts based clinical stage biopharmaceutical company Acceleron Pharma Inc. has announced its intent to offer and sell, subject to market and other conditions, $150 million of the company’s common stock in an underwritten public offering. Acceleron intends to use net proceeds from the offering partly to conduct clinical trials…

Researchers from Duke University have succeeded in treating an adult mouse model of Duchenne muscular dystrophy using the gene editing system CRISPR — the first successful treatment of a genetic disease in an adult animal utilizing a method that has the potential to be used in humans. In spite of recent advances…

Impaired cellular energy metabolism has been widely reported as a key element in dystrophin-deficient muscle degeneration, one of the most significant aspects of Duchenne Muscular Dystrophy (DMD). In a review entitled “Metabogenic and Nutriceutical Approaches to Address Energy Dysregulation and Skeletal Muscle Wasting in Duchenne Muscular Dystrophy,” the authors present…

UT Southwestern has been awarded a $7.8 million, five-year grant to establish the Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, with a special focus on inherited Duchenne muscular dystrophy (DMD). “UT Southwestern is the perfect environment for the Wellstone Center. We can merge cutting-edge science with clinical application,” said Dr.

Throughout 2015, Muscular Dystrophy News covered the latest developments in science, research, and advocacy news in muscular dystrophy. As the year comes to an end, here are the 10 articles most widely read by Muscular Dystrophy News readers, each with a brief summary of the developments that made them of such…

Researchers have identified two promising biomarkers for the noninvasive monitoring of Duchenne muscular dystrophy (DMD) disease progression or regression, although more research is necessary. The paper, titled “Comparative mass spectrometric and immunoassay-based proteome analysis in serum of Duchenne muscular dystrophy patients,” was published in Proteomics-Clinical Applications. Duchenne muscular dystrophy…

SOM Biotech announced the start of its first crowdfunding campaign to initiate new R&D projects into rare pediatric diseases, including Duchenne muscular dystrophy (DMD). The company, headquartered at the Barcelona Science Park (PCB), aims to raise at least $300,000 to be divided evenly among five rare childhood diseases: DMD, cystic fibrosis, Gaucher’s disease (…