The American Society of Gene and Cell Therapy (ASGCT), the primary professional body for gene and cell therapy, recently produced a guide on therapeutic gene editing to inform policy-makers, patient advocates, and the general public about the technique.
Articles by Özge Özkaya, PhD
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”.
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.
Resolaris, a potential intravenous protein therapy developed by aTyr Pharma for the treatment of rare myopathies is generally safe and well tolerated in patients with Facioscapulohumeral Muscular Dystrophy (FSHD) according to results presented at the 21st International Congress of the World Muscle Society.
A gene called CD40, which is an important modulator of the immune system, may determine the severity of Duchenne muscular dystrophy (DMD) according to a study published in The American Journal of Human Genetics. This finding suggests that drugs targeting CD40 may improve the symptoms of DMD.
People with Duchenne muscular dystrophy are most anxious and worried about their condition when they have to transition to using a wheelchair or a respirator due to the progression of their condition, found a study published in the International Journal of Qualitative Studies on Health and Well-being.
Researchers from Saudi Arabia demonstrated that a neurological panel assay that they developed, offers a high diagnostic yield for limb girdle muscular dystrophy (LGMD) and other myopathies.
In a recent article titled “Therapeutic Strategy for Heart Failure in Becker Muscular Dystrophy” and published in the International Heart Journal, Dr. Koichi Kimura of the University of Tokyo and colleagues present a review of the current strategies and suggest treatment options to treat heart failure in BMD.
Replenishing levels of a molecule naturally occurring in the body called NAD+ could restore muscle function according to scientists at the University of Pennsylvania.