News

CureDuchenne Responds to FDA Decision on Drug for Treatment of Duchenne Muscular Dystrophy

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…

DMD Exon Skipping Therapy Not Ready for Approval, FDA Says

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…

New Robotic Glove May Help MD Patients Retain Hand Movement

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…

Duchenne Muscular Dystrophy Drug to Be Developed by Grünenthal and Akashi Therapeutics

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…

First DMD Patients Enroll in Phase 2 Study of FibroGen’s FG-3019

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…

Young Duchenne MD Patients with Mild Arrhythmia Not Seen to Be at High Heart Risk

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 …