News

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…

A new smart rehabilitation device called EsoGlove developed by a research team from the National University of Singapore (NUS) is designed to assist patients who have lost hand function due to injuries or nerve-related disorders such as muscular dystrophy and stroke. NUS scientists say the EsoGlove is an improvement…

A group of international researchers has published a review of clinical trials for a drug that shows significant potential as a therapy for people who suffer from Duchenne muscular dystrophy (DMD). The study, published in the European Neurological Review, is titled “Idebenone as a Novel Therapeutic Approach…

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…

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…