Duchenne muscular dystrophy (DMD) is a progressive muscle disorder that causes the loss of both muscle function and independence. DMD is perhaps the most prevalent of the muscular dystrophies and is the most common lethal genetic disorder diagnosed during childhood today. Each year, approximately 20,000 children worldwide are born with DMD (one of every 3,500 male children).
Novo Biosciences Inc., has achieved several major milestones in its mission of bringing its breakthrough drug candidate, trodusquemine, to market as a potential regenerative medicine treatment for heart disease and Duchenne muscular dystrophy.
Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have implications for optimizing gene therapies for other diseases.
Patients with Duchenne muscular dystrophy have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.
Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.
Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease.
Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.
Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects.
Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense of touch.
The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions.
Heart and lung complications are responsible for much of the morbidity and mortality associated with Duchenne muscular dystrophy (DMD).
To help patients with muscle disorders, scientists at The University of Texas Health Science Center at Houston (UTHealth) have engineered a new stem cell line to study the conversion of stem cells into muscle. Findings appeared in Cell Reports.
Myotubular myopathy is a severe genetic disease that leads to muscle paralysis from birth and results in death before two years of age. Although no treatment currently exists, researchers from the University of Geneva, Switzerland, - working in collaboration with the University of Strasbourg, France, - have identified a molecule that not only greatly reduces the progression of the disease but also boosts life expectancy in animal models by a factor of seven.
Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.
Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patient's immune system is encountering for the first time.
Researchers in the United States have shown that genetically caused muscular dystrophy in dogs could be corrected using genetic editing tools. Muscular dystrophy is one of the most common fatal genetic conditions seen in children and is also seen in dogs
Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004).
The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.
Duchenne muscular dystrophy is one of the most common congenital diseases in the world, affecting one in 3,500 Canadian males. DMD is caused by mutations in the dystrophin gene that results in progressive muscle degeneration and there are currently no effective treatments for DMD.
A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in children and young adults with Duchenne muscular dystrophy, according to preliminary research presented at the American Heart Association's Basic Cardiovascular Sciences Scientific Sessions, a premier global exchange of the latest advances in basic cardiovascular science.
Insilico Medicine a leader in artificial intelligence for drug discovery, biomarker development and aging research, announced a research collaboration agreement with A2A Pharmaceuticals, Inc. A2A is a biotechnology company headquartered in New York and focused on development of novel drugs for unmet needs in oncology, drug resistant bacterial infections, and other life threatening diseases.