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UrduOver the past year, Children’s Hospital Los Angeles has been one of the highest-volume centers in the nation for Elevidys gene therapy. So far, CHLA has treated 10 boys with Duchenne muscular dystrophy with this gene transfer therapy—among the most in the U.S.Leading these efforts at CHLA is Leigh Ramos-Platt, MD, Director of the Neuromuscular Disorders Center in the Neurological Institute at Children’s Hospital Los Angeles, which includes one of the largest programs in California for Duchenne.
Tag: Duchenne Muscular Dystrophy
New gene therapy approach shows promise for Duchenne muscular dystrophy
Indiana University School of Medicine researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for people with Duchenne muscular dystrophy (DMD).
As FDA Expands Approval of a New Gene Therapy to Treat Duchenne Muscular Dystrophy, a CHLA Expert Weighs in
After previously approving its use for a limited population, the U.S. Food and Drug Administration has now approved Elevidys, a gene therapy designed to treat Duchenne muscular dystrophy (DMD), for a broader range of child patients.
Children’s Hospital Los Angeles Neurology Expert Available to Discuss FDA-Approved Gene Therapy Label Expansion for Patients with Duchenne Muscular Dystrophy
June 20, 2024 (Los Angeles) — Neurologist Leigh Ramos-Platt, MD, Medical Director of the Muscular Dystrophy Association Neuromuscular Clinic at Children’s Hospital Los Angeles (CHLA), is available to comment on the FDA expanded approval of gene therapy for patients with…
Lurie Children’s Hospital First in Illinois to Treat Duchenne Muscular Dystrophy with FDA-Approved Gene Therapy
On 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.
Jerry R. Mendell, MD, Receives 2023 Best Abstract Award for DMD Gene Therapy Research
The American Neuromuscular Foundation, is excited to announce Jerry R. Mendell, MD, as the recipient of the 2023 Best Abstract Award, for his abstract titled, “Long-Term Safety and Efficacy in Patients With Duchenne Muscular Dystrophy 4 Years Post-Treatment With Delandistrogene Moxeparvovec in a Phase 1/2a Study.”
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.
Scientists studying fatal muscle wasting disease make significant discovery
New research has revealed Duchenne muscular dystrophy (DMD) begins much earlier in cells destined to become muscle fibres, known as myoblasts.
Blocking an Ion Channel Improves Muscle Function and Survival in Mice with Severe Duchenne Muscular Dystrophy
Researchers at Johns Hopkins Medicine report that an experimental drug first developed to treat kidney disease prolongs survival and improves muscle function in mice genetically engineered to develop a severe form of Duchenne muscular dystrophy (DMD).
Piezo1 Possible Key to Supporting Muscle Regeneration in Duchenne Muscular Dystrophy
Tracing the impact of a single protein, Piezo1, Penn researchers found that restoring it in muscles affected by Duchenne muscular dystrophy could improve their ability to heal efficiently
Protein Deficiency Impairs Muscle Clock, Mitochondrial Function in Muscular Dystrophy
Article title: Dystrophin deficiency disrupts muscle clock expression and mitochondrial quality control in mdx mice Authors: Justin P. Hardee, Marissa K. Caldow, Audrey S.M. Chan, Stuart K. Plenderleith, Jennifer Trieu, Rene Koopman, Gordon S. Lynch From the authors: “These findings suggest that…
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.
Harrington Discovery Institute Announces Award Recipients in New UK Rare Disease Program
Announcement of the 2021 Harrington UK Rare Disease Scholar Award recipients, their organizations and fields of research:
Pietro Fratta, MD, PhD – University College London
Gene Therapy for Kennedy’s Disease, a rare neuromuscular disease
Angela Russell, DPhil – University of Oxford
New Drugs for the Treatment of Duchenne Muscular Dystrophy
Helen Waller-Evans, DPhil – Cardiff University
Novel Inhibitors to Treat Multiple Lysomal Storage Disorders, causes of widespread organ damage
Wyatt Yue, PhD – University of Oxford
Inhibitors of Primary Hyperoxaluria, a cause of kidney failure
Haiyan Zhou, MD, PhD – University College London
Novel Therapy for SPTLC1-Related Hereditary Sensory Neuropathy, a cause of shooting pain
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.
Encouraging First Report of Systemic Delivery of Micro-dystrophin Gene Therapy in Children with Duchenne Muscular Dystrophy
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.
Mouse Study Looks at Role of Transmembrane Transporter in Duchenne Muscular Dystrophy
Article title: Isoform-specific Na,K-ATPase and membrane cholesterol remodeling in motor endplates in distinct mouse models of myodystrophy Authors: Violetta V. Kravtsova, Elena V. Bouzinova, Alexander V. Chibalin, Vladimir V. Matchkov, Igor I. Krivoi From the authors: “Altogether, the α2 Na,K-ATPase and…
Releasing Brakes: Potential New Methods for Duchenne Muscular Dystrophy Therapies
Testing of small molecules in mouse models of Duchenne muscular dystrophy shows promise for restoration of muscle structure and function.
Duchenne Muscular Dystrophy Diagnosis Improved by Simple Accelerometers
Testing for Duchenne muscular dystrophy can require specialized equipment, invasive procedures and high expense, but measuring changes in muscle function and identifying compensatory walking gait could lead to earlier detection. This week in Chaos, researchers present a relative coupling coefficient, which can be used to quantify the factors involved in the human gait and more accurately screen for the disorder. They measured movements of different parts of the body in test subjects, viewing the body as a kinematic chain.
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).