Summit Therapeutics has enrolled its first U.S. patients in its proof-of-concept Phase 2 clinical trial of ezutromid in patients with Duchenne muscular dystrophy (DMD). The study is actively recruiting patients. “Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow…
News
Researchers have found that certain cognitive functions, specifically those requiring sequential processing of auditory and visual information, do not improve with age in patients with Duchenne muscular dystrophy (DMD). The study, “Profile of cognitive function in adults with duchenne muscular dystrophy,” was published in the journal Brain and…
Researchers from Australia recently reviewed experimental and clinical data from multiple research projects and clinical trials in an attempt to determine whether nutraceutical supplements could benefit or prolong the health of Duchenne MD patients. The findings suggest that some of these alternative products could be a valuable complementary therapy for…
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”.
News
Elevated Protein Improves Muscle, Pulmonary Dysfunction in Duchenne Muscular Dystrophy Mouse Models
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…
Recent Posts
- A tribute to a friend who taught us how to love, lead, and show up for others
- Patient-led group launches network of clinics to improve LGMD care
- To me, a wheelchair is a symbol of improved function, not disability
- When school is out, my sons with DMD get into their summer groove
- Upsher-Smith launches new DMD support websites for Kymbee treatment