Margarida Azevedo, MSc,  —

More than a dozen children in the Tampa, Florida area with muscular dystrophy, Down’s syndrome, and other neuromuscular disorders will soon be getting new and specially adapted bicycles donated by the ACMG Foundation for Genetic and Genomic Medicine. The 17 young recipients are from the local chapter of the Muscular Dystrophy Association (MDA) and …

Regenerative medicine is a branch of research that deals with the process of replacing, engineering or regenerating human cells, tissues, or organs to restore or establish normal function. But a major challenge in this field has been delivering treatments to control cellular behavior. One possible strategy to overcome this problem is to…

Catabasis Pharmaceuticals, Inc., recently announced that the first part of its MoveDMD clinical trial, a Phase 1/2 study of CAT-1004 in the treatment of Duchenne muscular dystrophy (DMD), has received positive top-line results, and it will soon begin the study’s second phase. “We are pleased with these results and believe that…

Duke University graduate student Mark Juhas is engineering a highly functional skeletal muscle tissue that has shown great promise and might constitute a valuable model for treatment of muscular dystrophy and other disorders, such as myositis, the inflammation and deterioration of the muscle tissue. According to the National Institute of Neurological…

Lochte Insurance Agency is organizing a charity drive to raise awareness and support for the San Antonio Muscle Walk on April 9, an annual event sponsored by the Muscular Dystrophy Association (MDA). The company’s goal is to pay for one child to attend the MDA Camp. The Lochte agency is…

Debra Miller, CEO and founder of CureDuchenne, a nonprofict dedicated to researching cures for Duchenne muscular dystrophy, recently released a statement following the U.S. Food and Drug Administration’s (FDA) decision to send a Complete Response Letter to BioMarin for its exon-skipping drug drisapersen (Kyndrisa). Duchenne muscular dystrophy, a devastating disease that…

BioMarin Pharmaceutical, Inc., announced that a Complete Response letter has been issued by the U.S. Food and Drug Administration (FDA) to the company’s New Drug Application (NDA) for Kyndrisa (drisapersen) for the treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. Complete Response Letters are issued to…

The Grünenthal Group, an international research and development pharmaceutical company, announced it is developing its Duchenne muscular dystrophy (DMD) medicine HT-100 in partnership with Akashi Therapeutics, a clinical stage biopharmaceutical company that focuses on the development of therapies for DMD and which until now has been funded entirely by DMD patient foundations. HT-100…

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…