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).
Researchers have developed an experimental model of severe Duchenne muscular dystrophy that offers insight into the disease.
With a new CRISPR gene-editing methodology, scientists from the University of Illinois at Urbana-Champaign inactivated one of the genes responsible for an inherited form of amyotrophic lateral sclerosis - a debilitating and fatal neurological disease for which there is no cure.
Researchers identified a group of small molecules that may open the door to developing new therapies for Duchenne muscular dystrophy, an as-yet-uncured disease that results in devastating muscle weakening and loss.
Duchenne muscular dystrophy is the most common type of muscular dystrophy, affecting more than 10,000 males at birth per year in the United States with severe physical disability, chronic wasting and muscle deterioration.
Duchenne type muscular dystrophy is the most common hereditary muscular disease among children, leaving them wheelchair-bound before the age of twelve and reducing life expectancy.
PerkinElmer, Inc., a global leader committed to innovating for a healthier world, today announced that it is providing the newborn screening assay for Parent Project Muscular Dystrophy’s Newborn Screening Pilot for Duchenne Muscular Dystrophy (Duchenne).
Horizon Discovery today announced that it will provide access to a novel base editing technology licensed from Rutgers for exclusive use in therapeutic, diagnostic and services applications.
A Rutgers-led team may have found the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease, the leading cause of death in patients living with the disease.
A new therapeutic being tested by University of Alberta researchers is showing early promise as a more effective treatment that could help nearly half of patients with Duchenne muscular dystrophy.
A new clinical trial conducted at The Ohio State University Wexner Medical Center found a cost-effective generic medication works just as well as a more expensive drug in preserving cardiovascular function in boys with Duchenne muscular dystrophy (DMD).
Duchenne muscular dystrophy (DMD) is a rare but devastating genetic disorder that causes muscle loss and physical impairments.
Researchers from Tufts University and the Chinese Academy of Sciences have developed a novel method to effectively deliver the CRISPR/Cas9 (clustered regularly interspaced short palindromic repeat (CRISPR) associated protein 9) gene editing tools into the liver for genetic studies.
The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy.
A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a major role in combating heart failure related to Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children.
Muscle decline caused by aging and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research shows.
A study published today in PLOS ONE discovered the origin of severely disfiguring masses of cells, called neurofibromas, that gradually develop throughout the skin of patients afflicted with Neurofibromatosis 1.
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.