The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) favors the renewal of the conditional marketing authorization of PTC Therapeutics’ Translarna (ataluren), an investigational drug in development for ambulatory patients 5 years and older, who have nonsense mutation Duchenne muscular dystrophy…
Women who received their initial risk assessment of being carriers of Duchenne muscular dystrophy (DMD) before 2003 should be reassessed in order to provide them with the right counseling about the risks of having a child with DMD and developing cardiomyopathy, according to a study published in the scientific journal Neurology Genetics.
Correction of scoliosis, or spine deformity with surgery could improve function, sitting balance, and quality of life in Duchenne muscular dystrophy (DMD) patients, according to a review article titled “Duchenne muscular dystrophy: the management of scoliosis”.
Researchers at the University of California investigated elevated levels of the protein sarcospan (SSPN) on skeletal muscle and pulmonary dysfunction in mice with Duchenne muscular dystrophy (DMD) and found notable improvements in disease symptoms. The study, “High levels of sarcospan are well tolerated and act as a sarcolemmal stabilizer to…
Heart complications in patients with Duchenne muscular dystrophy (DMD) are associated with shortening of telomeres, the protective DNA sequences at the ends of chromosomes, resulting from the lack of dystrophin (the hallmark of DMD), according to a new study. The study, “Telomere Shortening And Metabolic Compromise Underlie Dystrophic Cardiomyopathy,” was…
Actelion and ReveraGen BioPharma have entered an agreement in which Actelion has obtained the exclusive option to in-license ReveraGen’s lead compound vamorolone (VBP15) for the treatment of Duchenne muscular dystrophy (DMD) at two different development stages. Vamorolone is a novel compound with the potential to preserve muscle function…
T1 mapping, a cardiac magnetic resonance (CMR) imaging technique, can serve as a useful biomarker for detecting myocardial fibrosis and poorer heart function in young patients with Duchenne muscular dystrophy (DMD) and other cardiomyopathies, according to a new study. The study, “Native T1 Values Identify Myocardial Changes And Stratify Disease Severity In…
Two new studies that can explain more than half of the variability in disease progression seen in Duchenne muscular dystrophy (DMD) have recently been published. The results have important implications for the design of new and effective clinical trials testing the efficacy of potential DMD drugs.
Patients with Duchenne muscular dystrophy (DMD) who carry a variant in the CD40 gene, which encodes a protein of the immune system, have loss of ambulation at an earlier age, according to a study published in the American Journal of Human Genetics. The study, “Association Study Of Exon…
Parent Project Muscular Dystrophy (PPMD) has designated the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA its 12th Certified Duchenne Care Center. PPMD is a nonprofit that works to serve the Duchenne muscular dystrophy (DMD) community through researching new treatments and demanding the best care for patients. Its…
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