Marathon Pharmaceuticals' pricing of a drug to treat genetic muscle deterioration in about 15,000 Americans, mostly boys, is raising concerns in Congress where lawmakers repeatedly have challenged drug companies.
Feb 13, 2017
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(HealthDay) —Emflaza (deflazacort) has been approved by the U.S. Food and Drug Administration to treat Duchenne muscular dystrophy in people five years and older, the agency said Thursday in a news release.
Feb 9, 2017
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A newly discovered mutation in the INPP5K gene, which leads to short stature, muscle weakness, intellectual disability, and cataracts, suggests a new type of congenital muscular dystrophy. The research was published in the ...
Feb 9, 2017
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Researchers from Massachusetts General Hospital (MGH) led a large, international research team that has identified gene mutations associated with a rare congenital condition involving the absence of a nose and often accompanied ...
Jan 10, 2017
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Duchenne muscular dystrophy (DMD), a progressive muscle disease affecting one in 3800-6300 live male births and leads to ambulatory loss, respiratory problems, cardiomyopathy, and early death of patients in their 20s or 30s. ...
Jan 3, 2017
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The Food and Drug Administration on Friday approved the first treatment for children and adults with spinal muscular atrophy, a rare genetic disorder marked by progressive muscle weakness that's the most common genetic cause ...
Dec 23, 2016
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Scientists are using "gene scissors" to cut off the code of a defective gene that results in progressively weaker muscles and death in Duchenne muscular dystrophy and replace it with a synthetic code they hope will one day ...
Dec 19, 2016
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A team of scientists has uncovered details of the cellular mechanisms that control the direct programming of stem cells into motor neurons. The scientists analyzed changes that occur in the cells over the course of the reprogramming ...
Dec 8, 2016
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In 2013, Zoe Harting became the first baby in the world to receive an experimental drug that her doctors hoped would save the lives of thousands of infants like her.
Dec 6, 2016
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Infants as young as five weeks old with the most severe form of spinal muscular atrophy (SMA) - a leading genetic cause of infant mortality - can be treated safely with nusinersen. This investigational treatment slowed progression ...
Dec 6, 2016
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