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Muscular Dystrophy News
June 17, 2019

Top Headlines
 

CRISPR Halts Duchenne Muscular Dystrophy Progression in Dogs

Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a new study that provides a strong indication ... read more

'CRISPR-Gold' Fixes Duchenne Muscular Dystrophy Mutation in Mice

Scientists have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne ... read more

Gene Transfer Corrects Severe Muscle Defects in Mice With Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called ... read more

Study Could Herald New Treatment for Muscular Dystrophy

New research has shown that the corticosteroid deflazacort is a safe and effective treatment for Duchenne muscular dystrophy. The findings could pave the way for first US-approved treatment for the ... read more
Latest Headlines
updated 12:46pm EDT

Earlier Headlines
 

Worm Study Sparks Hope for Slowing Muscle Decline

Muscle decline caused by ageing and certain diseases could be dramatically slowed by stopping a chain reaction that damages cells, new research ... read more

Lithium Boosts Muscle Strength in Mice With Rare Muscular Dystrophy

Researchers have found that lithium improves muscle size and strength in mice with a rare form of muscular dystrophy that causes weakness in the shoulders and hips. The findings could lead to a drug ... read more

Novel Protein Degradation Pathway

A research team how a type of protein that is embedded in the inner nuclear membrane clears out of the system once it has served its ... read more

Muscle Gene Mutations Implicated in Human Nasal/sinus Cancer

By sequencing the entire genomes of tumor cells from six people with a rare cancer of the nose and sinus cavity, researchers report they unexpectedly found the same genetic change -- one in a gene ... read more

Stem Cell Researchers Develop Promising Technique to Generate New Muscle Cells in Lab

To help patients with muscle disorders, scientists have engineered a new stem cell line to study the conversion of stem cells into ... read more

A Molecule for Fighting Muscular Paralysis

Myotubular myopathy is a severe genetic disease that leads to muscle paralysis. Although no treatment currently exists, researchers have identified a molecule that not only greatly reduces the ... read more

Investigational Steroid Mirrors Prednisone's Benefits While Taming Its Side Effects

A head-to-head trial comparing the decades-old steroid, prednisone, and a promising new steroid, vamorolone, finds both act on the same key set of genetic pathways involved in controlling ... read more

Duchenne Muscular Dystrophy: How Muscle Cells Journey to the Dark Side

Answers to treating muscular dystrophies could lie in better understanding muscle repair -- which resembles a delicate cellular dance choreographed by special cells called fibro-adipogenic ... read more

Diseased Heart Muscle Cells Have Abnormally Shortened Telomeres

People with a form of heart disease called cardiomyopathy have abnormally short telomeres in heart muscle cells responsible for contraction, according to a new ... read more

Stabilizing Dysferlin-Deficient Muscle Cell Membrane Improves Muscle Function

In a head-to-head trial between the conventional glucocorticoid, prednisolone, and a modified glucocorticoid, vamorolone, in experimental models of LGMD2B, vamorolone improved dysferlin-deficient ... read more

Muscle Stem Cells Derived from Teratomas

Researchers have developed a process to regenerate skeletal muscle cells in mice with muscular ... read more

Dietary Supplement Increases Muscle Force by 50% Percent in the Duchenne Muscular Dystrophy Mouse Model

A dietary supplement derived from glucose increases muscle-force production in the Duchenne muscular dystrophy (DMD) mouse model by 50 percent in ten days, according to a new study. These results ... read more

Upgrading the Toolbox for Duchenne Muscular Dystrophy Research With a New Rabbit Model

Research to improve our understanding of Duchenne muscular dystrophy (DMD), and the development of new therapies, has previously relied on mouse models. However, physiological differences between the ... read more

Tamoxifen and Raloxifene Slow Down the Progression of Muscular Dystrophy

Steroids are currently the only available treatment to reduce the repetitive cycles of inflammation and disease progression associated with functional deterioration in patients with muscular ... read more

Human 'Chimeric' Cells Restore Crucial Protein in Duchenne Muscular Dystrophy

Cells made by fusing a normal human muscle cell with a muscle cell from a person with Duchenne muscular dystrophy -- a rare but fatal form of muscular dystrophy -- were able to significantly improve ... read more

Patients Living Longer With Duchenne Muscular Dystrophy Pose New Challenge for Caregivers

Diagnostic and treatment advances are helping patients with Duchenne muscular dystrophy live into their 30s and beyond, raising challenges in such areas as education, vocation, levels of ... read more

Cardiac Cell Therapy for Heart Failure Caused by Muscular Dystrophy Also Improves Skeletal Muscle Function in Mice

Injections of cardiac progenitor cells help reverse the fatal heart disease caused by Duchenne muscular dystrophy and also lead to improved limb strength and movement ability, a new study shows. The ... read more

Researchers Illustrate How Muscle Growth Inhibitor Is Activated, Could Aid in Treating ALS

Researchers have identified how the inactive or latent form of GDF8, a signaling protein also known as myostatin responsible for limiting muscle, is ... read more

Skeletal Muscle Created from Stem Cells

Scientists have developed a new strategy to efficiently isolate, mature and transplant skeletal muscle cells created from human pluripotent stem cells, which can produce all cell types of the body. ... read more

Scientists Slow Progression of Fatal Form of Muscular Dystrophy

Researchers report that a new drug reduces fibrosis (scarring) and prevents loss of muscle function in an animal model of Duchenne muscular dystrophy ... read more

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