Category Archives: Muscular Dystrophy Treatment

Approximately 1 in every 3,600 male infants worldwide is affected by Duchenne muscular dystrophy an inherited condition that causes severe muscle weakness. At present, there is no specific treatment for the disease. But new research published in the journal Neurology suggests that certain drugs usually prescribed for erectile dysfunction may be effective. Duchenne muscular dystrophy (DMD) is caused by a defective gene for dystrophin a protein that helps maintain healthy muscles. Low levels or absence of dystrophin means the muscles lack nitric oxide a chemical that signals blood vessels to dilate during exercise so blood flow can increase. As a result of the way the gene is inherited, the condition primarily affects boys and young men. Onset of the disease usually occurs before the age of 6 years. As well as muscle degeneration, the condition can cause intellectual disability, congestive heart failure or irregular heart rhythm, back and chest deformities and respiratory disorders. Many individuals with DMD are treated with corticosteroids. This medication can help to slow muscle degeneration and reduce negative effects on the heart and lung. But the researchers of this study, including Dr. Ronald G. Victor of the Cedars-Sinai Heart Institute in Los Angeles, CA, note that … Continue reading →

By Steven Reinberg HealthDay Reporter WEDNESDAY, May 7, 2014 (HealthDay News) -- Drugs normally prescribed to treat erectile dysfunction in adult males may help boys who have a muscle disease called Duchenne muscular dystrophy, according to a new study. In the small study including just 10 boys with the disease, researchers found that the popular drugs Viagra and Cialis improved blood flow to the boys' weakened muscles. "Boys with Duchenne muscular dystrophy have a blood flow abnormality -- delivery of blood and oxygen to their muscles -- that does not increase the way it should during mild exercise," said lead researcher Dr. Ronald Victor, the associate director of the Cedars-Sinai Heart Institute in Los Angeles. Because the blood vessels do not enlarge in a normal way in Duchenne muscular dystrophy, muscles are starved for oxygen, according to Victor. Cialis and Viagra work by enlarging blood vessels, thus increasing blood flow, which is the same way these drugs help men with erectile dysfunction. The report was published online May 7 in the journal Neurology. Duchenne muscular dystrophy is a genetic disease that affects boys and young men. It causes a progressive loss of muscle function to the point where those who … Continue reading →

Drugs that treat erectile dysfunction may be effective for boys suffering from Duchenne muscular dystrophy (DMD), Medical News Today reported. Duchenne muscular dystrophy (DMD) affects approximately 1 in every 3,600 male infants worldwide. The inherited condition causes severe muscle weakness and primarily affects boys and young men. DMD can cause intellectual disability, congestive heart failure or irregular heart rhythm, back and chest deformities, and respiratory disorders. Corticosteroids which slow down muscle degeneration and reduce DMDs effects on the heart and lungs have been used as treatment for the condition, but 75 percent of patients are unable to endure these kinds of drugs. In order to find an alternative treatment for DMD, researchers studied the effects of two erectile dysfunction drugs, sildenafil (Viagra) and tadalafil (Cialis), on patients with the condition. The drugs help increase blood flow to the muscles by relaxing blood vessels, potentially helping to maintain overall muscle health. For the study, researchers measured the blood flow of a group of 10 boys between the ages of 8 and 13 who had DMD, as well as a control group of 10 healthy boys. The boys with DMD then took each drug two weeks apart and had their blood flow … Continue reading →

Contact Information Available for logged-in reporters only Newswise LOS ANGELES (May 7, 2014) Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children. Muscle weakness begins in early childhood, often causing deformity of the arms, legs and spine. Heart and respiratory muscles often begin to fail before children reach early teen years. Although steroid medications which often are not well tolerated and other therapies may ease symptoms and delay the most severe effects, no disease-specific treatment exists, and patients rarely survive into their 30s. But in this case study of 10 boys with Duchenne muscular dystrophy, also called DMD, a single dose of a drug often prescribed for erectile dysfunction or pulmonary hypertension corrected defective blood vessel mechanisms and restored blood flow to exercising muscles. The effects were immediate and dramatic, raising the question: If a single dose restores blood flow to muscle while the drug is in the patients system, could ongoing tadalafil administration provide … Continue reading →

Drugs that treat erectile dysfunction may be effective for boys suffering from Duchenne muscular dystrophy (DMD), Medical News Today reported. Duchenne muscular dystrophy (DMD) affects approximately 1 in every 3,600 male infants worldwide. The inherited condition causes severe muscle weakness and primarily affects boys and young men. DMD can cause intellectual disability, congestive heart failure or irregular heart rhythm, back and chest deformities, and respiratory disorders. Corticosteroids which slow down muscle degeneration and reduce DMDs effects on the heart and lungs have been used as treatment for the condition, but 75 percent of patients are unable to endure these kinds of drugs. In order to find an alternative treatment for DMD, researchers studied the effects of two erectile dysfunction drugs, sildenafil (Viagra) and tadalafil (Cialis), on patients with the condition. The drugs help increase blood flow to the muscles by relaxing blood vessels, potentially helping to maintain overall muscle health. For the study, researchers measured the blood flow of a group of 10 boys between the ages of 8 and 13 who had DMD, as well as a control group of 10 healthy boys. The boys with DMD then took each drug two weeks apart and had their blood flow … Continue reading →

PUBLIC RELEASE DATE: 7-May-2014 Contact: Sally Stewart sally.stewart@cshs.org 310-248-6566 Cedars-Sinai Medical Center LOS ANGELES (May 7, 2014) Cedars-Sinai Heart Institute researchers have found that a commonly prescribed drug restores blood flow to oxygen-starved muscles of boys with Duchenne muscular dystrophy, a genetic muscle-wasting disease that rarely is seen in girls but affects one in 3,500 male babies, profoundly shortening life expectancy. It is the most common fatal disease that affects children. Muscle weakness begins in early childhood, often causing deformity of the arms, legs and spine. Heart and respiratory muscles often begin to fail before children reach early teen years. Although steroid medications which often are not well tolerated and other therapies may ease symptoms and delay the most severe effects, no disease-specific treatment exists, and patients rarely survive into their 30s. But in this case study of 10 boys with Duchenne muscular dystrophy, also called DMD, a single dose of a drug often prescribed for erectile dysfunction or pulmonary hypertension corrected defective blood vessel mechanisms and restored blood flow to exercising muscles. "The effects were immediate and dramatic, raising the question: If a single dose restores blood flow to muscle while the drug is in the patient's system, could … Continue reading →

PUBLIC RELEASE DATE: 7-May-2014 Contact: Rachel Seroka rseroka@aan.com 612-928-6129 American Academy of Neurology MINNEAPOLIS A drug typically prescribed for erectile dysfunction or increased pressure in the arteries may help improve blood flow in the muscles of boys with Duchenne muscular dystrophy, according to a study published in the May 7, 2014, online issue of Neurology, the medical journal of the American Academy of Neurology. Duchenne muscular dystrophy is a progressive and fatal muscle disease affecting boys and young men that causes loss of muscle function. There is no specific treatment for the disease, which is genetic. Corticosteroids can slow muscle degeneration and help temper the effect on lung and heart function, but they have many side effects and more than a quarter of people with the disease cannot tolerate the drugs. In Duchenne muscular dystrophy, the body has low or none of the protein called dystrophin, which helps maintain healthy muscles. Muscles are deprived of the chemical nitric oxide, which signals blood vessels to relax during exercise, increasing blood flow. The drug tadalafil, which works downstream of nitric oxide, acts to dilate the blood vessels. For the study, 10 boys age 8 to 13 with Duchenne muscular dystrophy who were … Continue reading →

Cialis, normally used to treat erectile dysfunction in men, may offer promise for boys with a fatal muscle disorder. In a preliminary study published today in the journal Neurology, the drug appears to improve blood flow to the boys' muscles, just as it boosts blood flow to the penis of men with erectile dysfunction. The drug was given to 10 boys over a short period, so the results are far from conclusive. They do indicate that Cialis deserves more research as a treatment for the boys' disease, known as Duchenne muscular dystrophy. "We had really striking results," said Ronald Victor, a cardiologist at Cedars-Sinai Medical Center in Los Angeles, who led the work. Examining the boys' muscle cells after treatment, he said, "it looked like a complete correction of the blood flow abnormality." In Duchenne muscular dystrophy, boys who are apparently normal until age 4-8, suddenly start to go weak. In their 20s or sometimes earlier, their heart or diaphragm ?? both muscles ?? give out, and they die. The genetic glitch that causes the disease robs the body of its ability to produce a muscle protein called dystrophin, which provides structure to muscles and helps send cellular signals. Without … Continue reading →

The most common form of muscular dystrophy among adults is dystrophia myotonica type 1 (DM1), where approximately 1 in every 8000 is affected by the disease. The severity of the disease varies from mild forms to severe congenital forms. It is dominantly inherited and accumulates through generations, gaining increased severity and lowered age of onset. DM1 is characterized by accumulating toxic aggregates of ribonucleic acids (RNA) from a specific mutated gene. When this RNA, which contains thousands of CUG nucleotide repeats, builds up in the cell, it attracts several cellular proteins, including muscleblind 1 (MBNL1). This binding inhibits the normal function of MBNL1, which means that the cellular level of a number of specific proteins becomes deregulated and the disease develops. Enzyme characterization in muscular dystrophy patients The researchers work at the Department of Molecular Biology and Genetics and the Department of Biomedicine, Aarhus University, where they characterized an enzyme called DDX6, found in both normal cells and cells from muscular dystrophy patients. The enzyme constantly tries to disperse the toxic aggregates and release MBNL1 in cells from muscular dystrophy patients, which means that the protein can carry out its normal function. The enzyme is found in many different cell … Continue reading →

Alexis, with parents Katrina and Adrian, and sister Eva. Staff reporter Friday, May 2, 2014 9:08 AM A boy from Southwold will be one of the first to be helped by a new nurse funded by an authors charity. To send a link to this page to a friend, you must be logged in. Five-year-old Alexis Ruthven was six months old when he was diagnosed with Duchenne muscular dystrophy, a condition that causes muscle weakness. About one in 3,500 boys have the condition, and it rarely affects girls. Alexis, his parents Adrian and Katrina, and his two sisters Eva, two, and Beatrice, one, used to travel down to Great Ormond Hospital in London for an annual health review. Mr Ruthven said: Wed have to leave early and come back late. It would be a whole day and wed sometimes stay down there. It was such an upheaval at the best of times. Read the original: Southwold boy with muscular dystrophy is first to be helped by new nurse service … Continue reading →