The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving ...
Jul 25, 2016
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Johns Hopkins University biologists have found that a protein that plays a key role in the lives of stem cells can bolster the growth of damaged muscle tissue, a step that could potentially contribute to treatments for muscle ...
Jul 19, 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 have introduced the world's first infant exoskeleton designed to help children with spinal muscular atrophy, a degenerative illness. Weighing 12 kilos, the apparatus is made of aluminium and titanium, and is designed ...
Jun 9, 2016
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Fresh insights into how our cells control muscle development could aid understanding of muscular dystrophy and other inherited diseases.
Jun 2, 2016
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The average healthy man is 54 percent muscle by mass, but people with muscular dystrophy, an incurable, genetic condition, have almost no muscle at terminal stages of the disease. New research from The Rockefeller University ...
Jun 1, 2016
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Scientists at the University of Liverpool have discovered that muscle cells affected by muscular dystrophy contain high levels of an enzyme that impairs muscle repair. This finding provides a new target for potential drug ...
Jun 1, 2016
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Many pediatricians and pediatric subspecialists believe that their clinical care extends from treating ill children through end-of-life care. However, are pediatricians actually meeting the needs of families and their dying ...
May 27, 2016
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Federal health regulators will take more time to review a highly-contested drug for muscular dystrophy that has become a flashpoint in the debate over patient access to experimental medicine.
May 25, 2016
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Michigan State University researchers used an old-fashioned neurobiology technique to explore new avenues for treatments to reverse a late-onset neurodegenerative disease that robs men of the capacity to walk, run, chew and ...
May 6, 2016
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