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UrduOn March 27, 2024, Ann & Robert H. Lurie Children’s Hospital of Chicago treated its first patient with ELEVIDYS (delandistrogene moxeparvovec-rokl), the first gene therapy for Duchenne muscular dystrophy – a rare, genetic disease characterized by progressive muscle damage and weakness.
Tag: DMD
Could a Novel Small Molecule Slow or Reverse the Effects of Duchenne Muscular Dystrophy?
In a new study published in The FASEB Journal, investigators demonstrated the potential of a molecule that may help overcome some of the devastating symptoms of Duchenne muscular dystrophy (DMD), the most common life-limiting congenital neuromuscular disorder. The agent promotes the activity of AMP-activated protein kinase (AMPK), an important fuel-sensing enzyme that is present in all mammalian cells.
Dr. Nicolau Receives 2022 Development Grant from American Neuromuscular Foundation
The American Neuromuscular Foundation (ANF), is
excited to announce the 2022 Development Grant Recipient, Stefan Nicolau, MD, for his
research project “CRISPR/Cas9 correction of a common Duchenne muscular dystrophy (DMD)
deletion.” Dr. Nicolau is a research fellow at the Abigail Wexner Research Institute at
Nationwide Children’s Hospital in Columbus, OH.
New Gene Editing Strategies Developed For Duchenne Muscular Dystrophy
DALLAS – April 30, 2021 – 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. The approach, described online today in the journal Science Advances, could lead to a treatment for DMD and inform the treatment of other inherited diseases.
Digging Deep For Differences In Duchenne Muscular Dystrophy
DALLAS – Dec. 21, 2020 – A UT Southwestern research team has catalogued gene activity in the skeletal muscle of mice, comparing healthy animals to those carrying a genetic mutation that causes Duchene muscular dystrophy (DMD) in humans. The findings, published online recently in PNAS, could lead to new treatments for this devastating degenerative disease and insights into factors that affect muscle development.
Genetic Mutation Could Worsen Heart Function in Duchenne Muscular Dystrophy Patients
DALLAS – Nov. 4, 2020 – 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. The findings, published online recently in the Journal of the American Heart Association, could help doctors develop new strategies to preserve heart function in this population, potentially extending patients’ lives.
Scientists uncover a novel approach to treating Duchenne muscular dystrophy
Scientists at Sanford Burnham Prebys, 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. The study, published in EMBO Reports, reveals a promising new therapeutic approach for Duchenne muscular dystrophy (DMD), an incurable muscle-wasting condition.
Heart-Function Protein May Help Muscular Dystrophy Patients Live Longer
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
Cheaper Drug Just As Effective Protecting Heart in 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).