Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane.
UT Southwestern scientists successfully employed a new type of gene therapy to treat mice with Duchenne muscular dystrophy (DMD), uniquely utilizing CRISPR-Cas9-based tools to restore a large section of the dystrophin protein that is missing in many DMD patients.
A first participant was dosed at I-Motion, the pediatric clinical trial platform for neuromuscular diseases located at Trousseau hospital in Paris, as part of the gene therapy trial in Duchenne muscular dystrophy (DMD) conducted by Genethon.
As the name implies, induced pluripotent stem cells can become any type of cell in our body, and scientists have evidence that when they prompt them to become muscle progenitor cells they can help restore the sometimes debilitating muscle loss that happens with age.
Today, the U.S. Food and Drug Administration granted approval for Amondys 45 (casimersen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 45 skipping (Exons are pieces of DNA that provide information for making proteins in a person's genome).
A muscle fiber consists of just one cell, but many nuclei. A team at the MDC led by Professor Carmen Birchmeier has now shown just how varied these nuclei are.
Genethon, dedicated to designing and developing gene therapy products for rare diseases, received this Monday 30th of November the authorization from the ANSM, the French National Agency for Medicines and Health Products Safety, to start in France a multicentre international clinical trial for the treatment of Duchenne muscular dystrophy with product GNT 004.
A mutation in the gene that causes cystic fibrosis may accelerate heart function decline in those with Duchenne muscular dystrophy (DMD), a new study by UT Southwestern researchers suggests.
Clinical Trials on a Chip researchers plan to build and test common and rare disease models to help improve the clinical trial process.
A new drug offers hope for young boys with the progressive neuromuscular disease Duchenne muscular dystrophy (DMD) by potentially offering an alternative to high-dose glucocorticoids that have significant side effects.
Scientists at Sanford Burnham Prebys Medical Discovery Institute, Fondazione Santa Lucia IRCCS, and Università Cattolica del Sacro Cuore in Rome have shown that pharmacological (drug) correction of the content of extracellular vesicles released within dystrophic muscles can restore their ability to regenerate muscle and prevent muscle scarring (fibrosis).
Researchers at the Universities of Maynooth and Bonn discover a new connection in muscular dystrophy Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance.
Today, the U.S. Food and Drug Administration granted accelerated approval to Viltepso (viltolarsen) injection for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping.
Researchers from Nationwide Children's Hospital have published in JAMA Neurology results from the first four patients treated in the first clinical trial of systemic delivery of micro-dystrophin gene therapy in children with Duchenne muscular dystrophy (DMD) - and initial findings suggest that the therapy can provide functional improvement that is greater than that observed under the standard of care.
A joint program of UT Southwestern Medical Center and Children's Health has been approved as a Certified Duchenne Care Center by Parent Project Muscular Dystrophy, the nation's most comprehensive nonprofit organization focused on finding a cure for Duchenne muscular dystrophy.
When an actor is unable to perform in the theatre, an understudy--ideally one with some practice in the role--can take her place on stage.
Researchers have developed an experimental model of severe Duchenne muscular dystrophy that offers insight into the disease.
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.
In a study recently published in Nature Communications scientists at Okayama University describe the detailed molecular pathogenesis of muscular dystrophy-associated cardiomyopathy in mice lacking the fukutin gene (Fktn), the causative gene for Fukuyama muscular dystrophy.
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).