This morning, Sarepta Therapeutics released data on eteplirsen, its experimental drug for muscular dystrophy.
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Many types of muscular dystrophy can also reduce the efficiency of the heart muscle.
More than 70, 000 babies, children and adults in the UK have muscular dystrophy or a related condition.
Some types of muscular dystrophy shorten the person's lifespan, often by affecting the muscles associated with breathing.
On the other hand, an effective muscular dystrophy drug is likely to support a very high price.
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"SMA has often been misrepresented to the public as muscular dystrophy, " says Columbia University neurologist Darryl De Vivo.
One muscular dystrophy patient, Steve Ledbrook, described his local region, the south west, as a "desert" for treatment.
"This is a remarkably hopeful time, " says Valerie Cwik, chief medical and scientific officer at the Muscular Dystrophy Association.
For instance, Wyeth is developing a medicine that would treat muscular dystrophy, but might also help athletes bulk up.
Untreated mice developed debilitation muscular dystrophy, suffered severe weight loss and half died before reaching 12 weeks of age.
Many people who have muscular dystrophy will eventually need to use a wheelchair.
Direct injection is also being used in gene-therapy trials on patients with Parkinson's and on those with muscular dystrophy.
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They have to play some role in the decision whether to pay for research into medieval poetry or muscular dystrophy.
Last November, Pfizer and Children's Hospital Boston signed a one-year research agreement to collaborate on fish screening in muscular dystrophy.
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Matt is one of 13, 000 boys in the U.S. who have a rare form of muscular dystrophy known as Duchenne.
Edwina Hart will meet families affected by muscle disease at the Muscular Dystrophy Campaign's conference in Swansea on 13 October.
DOWN'S syndrome, muscular dystrophy and haemophilia may be among the best-known genetic diseases, but they are most certainly not alone.
Dr. Kunkel's laboratory discovered the gene causing Duchenne muscular dystrophy in 1986.
The new medicine, eteplirsen, is targeted at the 13% of those boys whose muscular dystrophy is caused by a certain type of mutation.
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He persuaded a patient group, Parent Project Muscular Dystrophy, to grant a small amount of cash for preclinical work on the PTC medicine.
Muscular Dystrophy (MD) has many variations and having an accurate diagnosis is key to managing the disease. (Learn more about types of MD).
Four charities including CP Sport, Hope Against Cancer, Muscular Dystrophy Campaign and Pulmonary Fibrosis Research will benefit from funds raised on the day.
The four main charities to benefit will be Nottingham Hospitals Charity for Pulmonary Fibrosis Research, Hope Against Cancer, Cerebral Palsy Sport and Muscular Dystrophy.
And Sarepta is developing other drugs to treat muscular dystrophy in a way that may lay the groundwork for the medicines in the future.
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It can be used to check an embryo does not carry any one of 50 different conditions including cystic fibrosis and Duchenne muscular dystrophy.
Dr Marita Pohlschmidt, of the Muscular Dystrophy Campaign, which has funded the Newcastle research, was confident it would lead to a badly needed breakthrough in treatment.
For years, researchers have looked for ways of treating genetic diseases like muscular dystrophy, hemophilia and cystic fibrosis by targeting the genetic defects that cause them.
Genetic disorders, such as cystic fibrosis, muscular dystrophy and haemophilia, are caused by mutant variations of normal genes which are unable to perform their proper function.
Contractures may also play a part in the development of scoliosis, a sideways curvature of the spine that further reduces lung efficiency in people who have muscular dystrophy.
She said a pill to prevent muscle loss would unfortunately not be able to change the genetic cause of muscular dystrophy, but might slow down the disease progression.
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