Zebrafish, roundworms, fruit flies, mice, rats, rabbits, dogs, cats, pigs, and monkeys provide steppingstones to clinical trials to evaluate new treatments for people. The value of animal studies continues, even after a new ...
Mar 30, 2018
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Gene therapies aim to cure severe, barely treatable monogentic diseases caused by a defect in a single gene. Medical hopes are correspondingly high. Some gene therapies have already been approved in Europe—for example, ...
Aug 2, 2022
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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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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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A team of University of Missouri researchers have found that targeting a synthetic molecule to a specific gene could help the severity of the disease Spinal Muscular Atrophy (SMA) – the leading genetic cause of infantile ...
Nov 21, 2011
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In 2016, Spinraza became a game-changer for spinal muscular atrophy (SMA) patients. It was the first FDA-approved treatment for the neurodegenerative disease, which is the leading genetic cause of infant death. The drug ...
Jul 13, 2022
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Until recently, babies and children with spinal muscular atrophy (SMA) lacked any type of treatment capable of stopping the neuromuscular disease from progressing. In SMA, damaged motor neurons in the spinal cord fail to ...
Aug 20, 2021
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Investigators at Nationwide Children's Hospital may have discovered a biological explanation for why low levels of oxygen advance spinal muscular atrophy (SMA) symptoms and why breathing treatments help SMA patients live ...
Aug 21, 2012
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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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(Medical Xpress) -- An abnormally low level of a protein in certain nerve cells is linked to movement problems that characterize the deadly childhood disorder spinal muscular atrophy, new research in animals suggests.
Apr 11, 2012
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