Duchenne Muscular Dystrophy News and Research

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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).

Phase 1 data reinforce safety profile of new drug for treating Duchenne muscular dystrophy

Patients with Duchenne muscular dystrophy (DMD) have few treatment options. Medications currently available or in development either target only a subset of DMD patients with a particular genetic mutation or cause significant side effects.

21 Feb 2019

Unique gene therapy approach paves new way to tackle rare, inherited diseases

Nonsense mutations are single-letter errors in the genetic code that prematurely halt the production of critical proteins. These unfinished proteins are unable to function normally, and nonsense mutations cause 10-15 percent of all inherited genetic diseases, including Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and polycystic kidney disease.

19 Feb 2019

Single CRISPR treatment can safely and stably correct genetic disease

Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease -- Duchenne muscular dystrophy (DMD) -- for more than a year in mice, despite observed immune responses and alternative gene editing outcomes.

18 Feb 2019

Study findings hold promise for children with DMD

Prednisone, the current standard of care used to treat kids with Duchenne muscular dystrophy (DMD), reduces chronic inflammation but has harsh side effects.

11 Feb 2019

Scientists develop new gene therapy that prevents axon destruction in mice

Nerve axons serve as the wiring of the nervous system, sending electrical signals that control movement and sense of touch.

18 Jan 2019

Researchers identify and overcome barrier in CRISPR gene editing to treat muscular dystrophy

The gene editing technique known as CRISPR is a revolutionary approach to treating inherited diseases. However, the tool has yet to be used to effectively treat long-term, chronic conditions.

9 Jan 2019

Home-based pulmonary function monitoring for teens with Duchenne muscular dystrophy

Heart and lung complications are responsible for much of the morbidity and mortality associated with Duchenne muscular dystrophy (DMD).

18 Dec 2018

Scientists develop new stem cell line to study conversion of stem cells into muscle

To help patients with muscle disorders, scientists at The University of Texas Health Science Center at Houston (UTHealth) have engineered a new stem cell line to study the conversion of stem cells into muscle. Findings appeared in Cell Reports.

13 Dec 2018

Researchers identify molecule to fight myotubular myopathy

Myotubular myopathy is a severe genetic disease that leads to muscle paralysis from birth and results in death before two years of age. Although no treatment currently exists, researchers from the University of Geneva, Switzerland, - working in collaboration with the University of Strasbourg, France, - have identified a molecule that not only greatly reduces the progression of the disease but also boosts life expectancy in animal models by a factor of seven.

19 Nov 2018

Urine exRNA may be source of biomarkers for muscular dystrophy

Massachusetts General Hospital researchers have found that extracellular RNA in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

26 Sep 2018

Stanford researchers show how to prevent immune response to gene therapy in mouse model

Stanford University School of Medicine researchers have demonstrated that gene therapy can be effective without causing a dangerous side effect common to all gene therapy: an autoimmune reaction to the normal protein, which the patient's immune system is encountering for the first time.

4 Sep 2018

Muscle dystrophy treatment in dogs with Crispr gene editing

Researchers in the United States have shown that genetically caused muscular dystrophy in dogs could be corrected using genetic editing tools. Muscular dystrophy is one of the most common fatal genetic conditions seen in children and is also seen in dogs

31 Aug 2018

Pfizer terminates clinical studies evaluating domagrozumab for treatment of Duchenne muscular dystrophy

Pfizer Inc. announced today that it is terminating two ongoing clinical studies evaluating domagrozumab (PF-06252616) for the treatment of Duchenne muscular dystrophy (DMD): a Phase 2 safety and efficacy study (B5161002) and an open-label extension study (B5161004).

30 Aug 2018

MDA announces 34 new grants totaling $9.9 million to accelerate neuromuscular research

The Muscular Dystrophy Association today announced the award of 34 new grants totaling more than $9.9 million for its summer round of funding. These new grants represent a continued commitment by MDA to fund groundbreaking research that will accelerate treatments and cures for the more than 40 diseases in its program.

24 Aug 2018

New project to combat DMD-related fibrosis receives major funding boost

Duchenne muscular dystrophy is one of the most common congenital diseases in the world, affecting one in 3,500 Canadian males. DMD is caused by mutations in the dystrophin gene that results in progressive muscle degeneration and there are currently no effective treatments for DMD.

17 Aug 2018

Protein known to drive nerve cell survival may protect children from Duchenne cardiomyopathy

A protein known to drive nerve cell survival in the brain and spinal cord might also protect failing hearts in children and young adults with Duchenne muscular dystrophy, according to preliminary research presented at the American Heart Association's Basic Cardiovascular Sciences Scientific Sessions, a premier global exchange of the latest advances in basic cardiovascular science.

2 Aug 2018

Top AI companies join hands to discover novel drugs for DMD

Insilico Medicine a leader in artificial intelligence for drug discovery, biomarker development and aging research, announced a research collaboration agreement with A2A Pharmaceuticals, Inc. A2A is a biotechnology company headquartered in New York and focused on development of novel drugs for unmet needs in oncology, drug resistant bacterial infections, and other life threatening diseases.

20 Jul 2018

Glucose-based dietary supplement increases muscle-force in Duchenne muscular dystrophy mouse

A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy mouse model by 50% in ten days, according to a study conducted by researchers from Université Laval's Faculty of Medicine and Centre hospitalier universitaire de Québec Research Centre-Université Laval.

30 Jun 2018

CRISPR technique reduces repetitive behavior in fragile X syndrome mouse models

Scientists have used CRISPR-Cas9 gene editing to lessen some autism symptoms in mice with a form of fragile X syndrome, the most common known single-gene cause of autism spectrum disorder.

25 Jun 2018

KFU medical departments seek to find treatments for hereditary syndromes

Newest results were showcased at the International Myology School in Moscow on 16th - 19th May 2018. KFU was represented by Junior Research Associate Mikhail Mavlikeev.

6 Jun 2018