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).
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.
A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy mouse model by 50% in ten days, according to a study conducted by researchers from Université Laval's Faculty of Medicine and Centre hospitalier universitaire de Québec Research Centre-Université Laval.
Scientists have used CRISPR-Cas9 gene editing to lessen some autism symptoms in mice with a form of fragile X syndrome, the most common known single-gene cause of autism spectrum disorder.
Newest results were showcased at the International Myology School in Moscow on 16th - 19th May 2018. KFU was represented by Junior Research Associate Mikhail Mavlikeev.
Researchers from Queen Mary University of London have developed new cell-based technologies which could help improve understanding of the muscle-wasting disease Duchenne muscular dystrophy (DMD) and test potential drugs for the disease.
Duo Zhang, PhD, a postdoctoral associate at Boston University School of Medicine, has received the highly competitive National Institutes of Health Pathway to Independence Award.
Zach Smith has Duchenne muscular dystrophy, a genetic disorder marked by progressive muscle degeneration. His lack of muscle control and being in a wheelchair made him a prime candidate for a computer-controlled exoskeleton arm.
Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy --a rare but fatal form of muscular dystrophy -- were able to significantly improve muscle function when implanted into the muscles of a mouse model of the disease.
Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy-;one of nine major types of muscular dystrophy that affects males-;live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of independence, personal relationships, emotional health, and intimacy.
The University of Plymouth will be continuing its research into the advancement of neuro-tumor treatments thanks to more than £100,000 from Great Ormond Street Hospital Children's Charity and Sparks, the medical research charity.
Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows.
Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of the 3,000 mutations that cause Duchenne muscular dystrophy (DMD) by making a single cut at strategic points along the patient's DNA, according to a study from UT Southwestern Medical Center.
Researchers have successfully created artificial human muscles in the laboratories that are tiny but fully functional. These small muscles actually respond to the nerve stimulations, says the latest study reporting this scientific breakthrough.
The commissioner of the Food and Drug Administration questions whether the right financial incentives are in place for drugmakers who develop orphan drugs for rare diseases.