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 at UBC’s School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.
Researchers at the University of British Columbia's School of Biomedical Engineering have discovered that an existing cancer drug could have potential as a treatment for muscular dystrophy.
In about a fifth of the cases of Parkinson’s disease, look to a small, malfunctioning protein in the lysosome as a risk factor, say University of Michigan researchers.
The development of messenger RNA (mRNA) vaccines against the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) has only increased the impetus in this field.
New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change in how the disease is treated.
In proof-of-concept experiments, Johns Hopkins Medicine scientists say they have successfully cultivated human muscle stem cells capable of renewing themselves and repairing muscle tissue damage in mice, potentially advancing efforts to treat muscle injuries and muscle-wasting disorders in people.
Researchers discuss the use of the highly efficient CRISPR/Cas technologies to identify viral genome sequences for biosafety purposes.
One protein, Piezo1, is key to marshalling muscle stem cells' unique shapes and response to injuries, but it is in low supply in those with Duchenne muscular dystrophy, according to a team at the Perelman School of Medicine at the University of Pennsylvania.
A clinical trial at UC Davis Health and six other sites showed that a cellular therapy offers promise for patients with late-stage Duchenne muscular dystrophy (DMD), a rare genetic disorder causing muscle loss and physical impairments in young people.
Glucocorticoid steroids improved muscle performance through distinct, sex-specific molecular mechanisms in mice, according to a Northwestern Medicine study published in the Journal of Clinical Investigation.
The drug industry, patient advocates, and congressional Republicans have all attacked federal officials' decision to decline routine Medicare coverage for a controversial Alzheimer's drug.
In a new study, the group of Johan Auwerx at EPFL's School of Life Sciences has made the first connection between muscular dystrophy and sphingolipids, a group of bioactive lipids. The study is published in Science Advances.
In this interview, News-Medical speaks to Dr. Jyoti K. Jaiswal about his latest research into gene therapies for limb-girdle muscular dystrophy 2B.
Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene known as C9ORF72 in a single-patient pilot study.
Many more adults with Duchenne muscular dystrophy (DMD) are living longer thanks to improvements in treatment, however international standards of care have not yet fully addressed the complex needs of these patients.
Children's Hospital Los Angeles cardiologist Michael Silka, MD, helped to pioneer the development of indications for the use of pacemakers and implantable defibrillators in young patients.
This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional dystrophin proteins, as described in the peer-reviewed journal Nucleic Acid Therapeutics.
In the era of "publish or perish," research results that disprove the hypothesis tested often go unpublished. In addition, many researchers who have been unable to reproduce results from other laboratories have found it difficult to publish their findings.
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced that the organization's Newborn Screening Pilot has successfully reached its completion, screening more than 36,000 babies born in New York State over the last two years.
Specialized cells that conduct electricity to keep the heart beating have a previously unrecognized ability to regenerate in the days after birth, a new study in mice by UT Southwestern researchers suggests.