A new treatment for Huntington's disease – a deadly brain disorder – has successfully completed first-in-human trials. The drug lowered levels of the harmful huntingtin protein in the spinal fluid of patients who took ...
Dec 14, 2017
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When the Food and Drug Administration approved the first drug for people with spinal muscular atrophy a year ago, clinicians finally had hope for improving the lives of patients with the rare debilitating muscular disease. ...
Dec 11, 2017
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Research from the University of Warwick indicates that most people are in favour of newborn screening for the potentially deadly condition spinal muscular atrophy (SMA). The study Newborn genetic screening for spinal muscular ...
Dec 5, 2017
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Glucocorticoids, a class of steroid hormone medications often prescribed to patients with Duchenne muscular dystrophy (DMD), offer long-term benefits for this disease, including longer preservation of muscle strength and ...
Nov 22, 2017
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After boys and young men with Duchenne muscular dystrophy received cardiac progenitor cell infusions, medical tests indicated that the patients' hearts appeared improved, results from a new study show. Patients in the study ...
Nov 15, 2017
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New research by the Murdoch Children's Research Institute (MCRI) has shown that the combined affected pregnancy rate of cystic fibrosis (CF), fragile X syndrome (FXS) and spinal muscular atrophy (SMA) is comparable to the ...
Nov 9, 2017
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In a recent paper published in the journal Skeletal Muscle, a Saint Louis University researcher reports success in identifying new drug targets that potentially could slow or halt the progression of a form muscular dystrophy, ...
Nov 7, 2017
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(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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