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).
The U.S. Food and Drug Administration today approved Emflaza (deflazacort) tablets and oral suspension to treat patients age 5 years and older with Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes progressive muscle deterioration and weakness.
Duchenne muscular dystrophy (DMD), a progressive muscle disease affecting one in 3800-6300 live male births and leads to ambulatory loss, respiratory problems, cardiomyopathy, and early death of patients in their 20s or 30s.
The Muscular Dystrophy Association today celebrated news of the U.S. Food and Drug Administration's decision to grant approval for nusinersen (brand name Spinraza), the first disease-modifying drug to treat the most common genetic cause of death in infants.
Researchers from the Cedars-Sinai Heart Institute and the Cedars-Sinai Department of Medicine are expanding their ongoing evaluation of a novel cell-based therapeutic candidate into the area of pulmonary arterial hypertension (PAH).
Researchers with funding from Fight for Sight have demonstrated that a new drug treatment for cystic fibrosis and Duchenne muscular dystrophy can override a genetic fault that causes choroideremia – a severe blinding disorder.
In September, the Food and Drug Administration approved Exondys, a controversial treatment for Duchenne muscular dystrophy based on tenuous data from just 12 patients.
Retinal activity in autism, ADHD and Duchenne muscular dystrophy
A new paper, co-written by faculty at Binghamton University, State University of New York, increases the understanding of Duchenne muscular dystrophy (DMD)—one of the most common lethal genetic disorders—and points to potential therapeutic approaches.
Nearly 1,000 boys in the New York Tri-State area have been diagnosed with Duchenne Muscular Dystrophy (DMD) and, until now, had to travel out of the state for comprehensive care.
Building on the results of a recent Cedars-Sinai Heart Institute study published just six months ago, the Department of Defense has awarded a $10 million grant to fund a cardiac cell therapy trial for patients diagnosed with a common but difficult-to-treat form of heart failure.
Using high-resolution electron microscopy, Columbia University Medical Center researchers have uncovered new details of the structure and function of an intracellular channel that controls the contraction of skeletal muscle.
The U.S. Food and Drug Administration today approved Exondys 51 (eteplirsen) injection, the first drug approved to treat patients with Duchenne muscular dystrophy (DMD).
New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings, which appear this month in the journal Neurology, could pave the way for first U.S.-approved treatment for the disease.
The U.S. Patent and Trademark Office has announced that it will grant a patent to MDI Biological Laboratory scientists Voot P. Yin, Ph.D., and Kevin Strange, Ph.D., and their collaborator Michael Zasloff, M.D., Ph.D., for use of the small molecule MSI-1436 to stimulate the repair and regeneration of heart tissue damaged by injuries such as a heart attack.
A discovery by Washington State University scientist Dan Rodgers and collaborator Paul Gregorevic could save millions of people suffering from muscle wasting disease.
A key gene enables the repair of injured muscle throughout life. This is the finding of a study in mice led by researchers at NYU Langone Medical Center and the University of Colorado at Boulder, and published online July 21 in Cell Reports.
Satellite cells are stem cells found in skeletal muscles. While transplantation of such muscle stem cells can be a potent therapy for degenerative muscle diseases such as Duchenne muscular dystrophy, these cells tend to lose their transplantation efficiency when cultured in vitro.
Although neurofibromatosis (NF) is not commonly discussed, it affects more than 2 million people worldwide.
Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug treatments for the disease, which currently has no cure.
Johns Hopkins researchers report they have inadvertently found a way to make human muscle cells bearing genetic mutations from people with Duchenne muscular dystrophy (DMD).