Impaired transport processes in neurons contribute to diseases such as amyotrophic lateral sclerosis (AML). Würzburg scientists have now identified key actors in these processes.
Mar 9, 2018
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They are many rare genetic diseases that strike perhaps only one in a million people. Often incurable, they can be profoundly debilitating and frequently life-threatening. Though each particular disease is rare, they number ...
Feb 28, 2018
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Children with later-onset spinal muscular atrophy (SMA) were more likely to show gains in motor function when treated with a new medication compared to children receiving a sham procedure, according to a study published today ...
Feb 15, 2018
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UCLA researchers have discovered the inner workings of a gene network that regulates the development of spinal motor neurons in the growing chicken and mouse embryo. The research also answers a long-standing question about ...
Feb 2, 2018
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Research led by The Ohio State University Wexner Medical Center to define the natural history of infantile-onset spinal muscular atrophy (SMA) has been "critical" to accelerate the development of effective therapies and hasten ...
Jan 9, 2018
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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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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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(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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