Setback in gene therapy for Duchenne muscular dystrophy as immune system emerges as key barrier
A new paper, published in Gene Therapy, raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy…
A new paper, published in Gene Therapy, raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy…
A groundbreaking study has shed light on the complex interactions between dystrophin, a protein critical to muscle stability, and its…
A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration….
A pilot study using a new screening tool shows promise for assessing neurobehavioral difficulties in children with Duchenne muscular dystrophy…
Moderate levels of physical activity and fitness may be linked to a reduced risk of amyotrophic lateral sclerosis (ALS) later…
he U.S. Food and Drug Administration expanded the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of…
A recent study has found that the SMCHD1 protein plays a key role in controlling how genes are processed, which…
The U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in…
The drug vamorolone (Agamree®) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has…
Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body…
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves…
New research published in JAMA recommends daily steroid doses for children with Duchenne muscular dystrophy (DMD), marking a significant change…
A cell therapy developed by the executive director of the Smidt Heart Institute stabilizes weakened muscles–including the heart muscle–in Duchenne…
Each year, about 20,000 children are diagnosed with Duchenne muscular dystrophy, a rare genetic condition that causes progressive muscle weakness…
In a new study, the group of Johan Auwerx at EPFL’s School of Life Sciences has made the first connection…
Many more adults with Duchenne muscular dystrophy (DMD) are living longer thanks to improvements in treatment, however international standards of…
Muscle stem cells enable our muscle to build up and regenerate over a lifetime through exercise. But if certain muscle…
New research from the University of Minnesota Medical School found mutations in a novel gene that may help identify patients…
A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy…
Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance….
A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in…
New research published in The Journal of Physiology today suggests that enhancing breathing via the brain may limit deficiencies in…