Tag: Muscular Dystrophy

Neurology

UTSW/Children's Health joint program approved as Certified Duchenne Care Center

Newsemia
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,...
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New drug provides hope for patients with Duchenne muscular dystrophy

Newsemia
The results from three clinical trials have shown that a new drug can successfully delay the progression of Duchenne muscular dystrophy. Source link...
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New discovery could improve therapies for Duchenne muscular dystrophy

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A new multi-institution study spearheaded by researchers at Florida State University and the University of California, Los Angeles suggests a tiny protein could play a...
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Researchers develop new method to boost efficiency of CRISPR gene editing in DMD

Newsemia
Scientists have developed a method to boost the efficiency of CRISPR gene editing in Duchenne muscular dystrophy (DMD), according to a study that could have...
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Unique gene therapy approach paves new way to tackle rare, inherited diseases

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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,...
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Single CRISPR treatment can safely and stably correct genetic disease

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Researchers at Duke University have shown that a single systemic treatment using CRISPR genome editing technology can safely and stably correct a genetic disease —...
Neurology

Scientists develop new gene therapy that prevents axon destruction in mice

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

New mouse model shows potential for rapid identification of promising muscular dystrophy therapies

Newsemia
A Massachusetts General Hospital research team has created a new mouse model of a common form of muscular dystrophy with the potential of rapidly distinguishing...
Neurology

Urine exRNA may be source of biomarkers for muscular dystrophy

Newsemia
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...

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