(HealthDay)—Gene replacement therapy is beneficial in spinal muscular atrophy type 1 (SMA1), and nusinersen is beneficial for infants with spinal muscular atrophy, according to two studies published online Nov. 1 in the ...
Nov 2, 2017
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Motor neurons are the nerves that send impulses to the muscles to generate movement. Damage of these neurons can cause very diverse diseases, for example spinal muscular atrophy in children or adult amyotrophic lateral sclerosis.
Nov 2, 2017
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Infants with the most severe form of spinal muscular atrophy (SMA) were more likely to show gains in motor function and were 47 percent more likely to survive without permanent assisted ventilation support when treated with ...
Nov 1, 2017
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The ability to cheaply and quickly sequence entire genomes is changing the way diseases are identified and treated. But it is also likely to change the way we make some of the most important and personal decisions of our ...
Aug 2, 2017
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Duchenne muscular dystrophy is a rapidly progressive disease that causes whole-body muscle weakness and atrophy due to deficiency in a protein called dystrophin. Researchers at the University of Missouri, National Center ...
Jul 27, 2017
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The Miami Project to Cure Paralysis, a Center of Excellence at the University of Miami Miller School of Medicine, in collaboration with Scholar Rock, a biotechnology company focused on discovering and developing drugs that ...
Jul 10, 2017
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Spinal muscular atrophy (SMA), a neurodegenerative disease that causes progressive muscle wasting and paralysis, may be partly due to abnormalities in the synapses that connect sensory neurons and motor neurons, according ...
May 15, 2017
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When Marathon Pharmaceuticals announced in February it would market a drug for treating Duchenne muscular dystrophy for US$89,000 a year, the negative reaction was so intense that the company immediately suspended the rollout. ...
Mar 17, 2017
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(HealthDay)—The American College of Obstetricians and Gynecologists (ACOG) recommends use of carrier screening for all women, according to two Committee Opinions published in the March issue of Obstetrics & Gynecology.
Feb 27, 2017
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Researchers working at Karolinska Institutet in Sweden and University of Southern Denmark have managed to produce short synthetic DNA analogues – oligonucleotides – that bind directly to the gene that is mutated in Huntington's ...
Feb 20, 2017
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