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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A puzzling question has lurked behind SMA (spinal muscular atrophy), the leading genetic cause of death in infants.
Feb 14, 2017
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Johns Hopkins researchers along with academic and drug industry investigators say they have identified a new biological target for treating spinal muscular atrophy. They report they have evidence that an experimental medicine ...
Jan 9, 2017
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A research team led by the University of Oxford has found a promising treatment for degenerative disease spinal muscular atrophy (SMA), a leading genetic cause of child death.
Sep 20, 2016
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Scientists and doctors know that the devastating disease spinal muscular atrophy (SMA) arises from a problem with both copies of the SMN1 gene, leading to a lack of the survival motor neuron (SMN) protein. But they don't ...
Jul 11, 2016
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Researchers at Iowa State University have found evidence that a "housekeeping" gene present in every cell of the body may have a link to male infertility.
Feb 3, 2016
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A tiny sliver of a person's DNA—several thousand times smaller than a typical gene—produces a molecule that has crucial influence over whether a person has any control over their muscles, according to a paper published ...
Dec 17, 2015
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In work involving several new generations of mouse model development, Jackson Laboratory (JAX) researchers have tested a therapeutic intervention for spinal muscular atrophy (SMA) that restores some function lost due to a ...
Oct 14, 2015
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In the decade since the genome was sequenced in 2003, scientists and doctors have struggled to answer an all-consuming question: Which DNA mutations cause disease?
Dec 18, 2014
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Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in a mouse model ...
Aug 7, 2014
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