News

Sarepta Therapeutics, Inc., announced that a U.S. Food and Drug Administration (FDA) advisory committee has been forced to postpone Friday’s scheduled hearing to review the company’s New Drug Application (NDA) for eteplirsen, a potential treatment of Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping. A severe winter storm starting…

Researchers have found that RNA sequencing is able to identify disease-causing genetic mutations in non-coding regions of the DNA. The technique can be used to aid in diagnosing muscular dystrophy (MD). The study, “RNAseq analysis for the diagnosis of muscular dystrophy,“ appeared in the journal Annals of Clinical and Translational…

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…

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 …