ScienceDaily: Muscular Dystrophy News
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Laminopathies: Key components in the disease mechanism identified
Laminopathies are hereditary diseases that affect mainly the muscle tissue. These diseases include for example Emery-Dreifuss Muscular dystrophy, dilated cardiomyopathy, limb-girdle muscular dystrophy and Hutchison-Gilford progeria syndrome.
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Firefly protein lights up degenerating muscles, aiding muscular-dystrophy research
Scientists have created a mouse model of muscular dystrophy in which degenerating muscle tissue gives off visible light.
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Why a hereditary anemia is caused by genetic mutation in mechanically sensitive ion channel
A genetic mutation that alters the kinetics of an ion channel in red blood cells has been identified as the cause behind a hereditary anemia, according to a new article.
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Genetically corrected stem cells spark muscle regeneration
Researchers have combined genetic repair with cellular reprogramming to generate stem cells capable of muscle regeneration in a mouse model for Duchenne muscular dystrophy.
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Cell discovery could hold key to causes of inherited diseases
Fresh insights into the protective seal that surrounds the DNA of our cells could help develop treatments for inherited muscle, brain, bone and skin disorders. Researchers have discovered that the proteins within this coating -- known as the nuclear envelope -- vary greatly between cells in different organs of the body.
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Experimental gene therapy treatment for Duchenne muscular dystrophy offers hope for youngster
Jacob Rutt is a bright 11-year-old who likes to draw detailed maps in his spare time. But the budding geographer has a hard time with physical skills most children take for granted -- running and climbing trees are beyond him, and even walking can be difficult. He was diagnosed with a form of muscular dystrophy known as Duchenne when he was two years old.
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Gene therapy: Editing out genetic damage
New design guidelines from researchers in Singapore simplify the development of targeted therapies for muscular dystrophy and other diseases.
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Scientists discover 'needle in a haystack' for muscular dystrophy patients
Scientists have identified a sequence in the dystrophin gene that is essential for helping muscle tissues function, a breakthrough discovery that could lead to treatments for the deadly hereditary disease. The researchers “found the proverbial needle in a haystack,” according to an expert.
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Quantum leap in gene therapy of Duchenne muscular dystrophy
For years, scientists have been working to find the key to restoring dystrophin, but they have faced many challenges. After careful evaluation of 22 dogs, researchers found that the new version of the micro-dystrophin gene not only reduced inflammation and fibrosis, it also effectively improved muscle strength.
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Tamoxifen ameliorates symptoms of Duchenne muscular dystrophy, study suggests
A new study has found that tamoxifen, a well-known breast cancer drug, can counteract some pathological features in a mouse model of Duchenne muscular dystrophy (DMD). At present, no treatment is known to produce long-term improvement of the symptoms in boys with DMD, a debilitating muscular disorder that is characterized by progressive muscle wasting, respiratory and cardiac impairments, paralysis, and premature death.
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Stem-cell approach shows promise for Duchenne muscular dystrophy
Researchers have shown that transplanting stem cells derived from normal mouse blood vessels into the hearts of mice that model the pathology associated with Duchenne muscular dystrophy (DMD) prevents the decrease in heart function associated with DMD.
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Drug that may help fight Duchenne muscular dystrophy discovered
Drugs are currently being tested that show promise in treating patients with Duchenne muscular dystrophy (DMD), an inherited disease that affects about one in 3,600 boys and results in muscle degeneration and, eventually, death.
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Another muscular dystrophy mystery solved; Scientists inch closer to a therapy for patients
In a new study, researchers have discovered the missing pieces to a muscular dystrophy puzzle that could ultimately lead to a therapy and, potentially, a longer lifespan for patients suffering from the disease.
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Common drug reverses common effect of becker muscular dystrophy
Researchers have found in an initial clinical trial that a drug typically prescribed for erectile dysfunction or pulmonary hypertension restores blood flow to oxygen-starved muscles in patients with a type of muscular dystrophy that affects males, typically starting in childhood or adolescence.
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Protein injection points to muscular dystrophy treatment
Scientists have discovered that injecting a novel human protein into muscle affected by Duchenne muscular dystrophy significantly increases its size and strength, findings that could lead to a therapy akin to the use of insulin by diabetics.