In an announcement that delighted the families of patients and surprised many pharmaceutical industry analysts, the biotechnology firms Biogen and Ionis Pharmaceuticals on Monday said they had decided to cut short a phase-3 ...
Aug 4, 2016
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A new study by a team of scientists from the University of Malta and the Institut de Génétique Moléculaire de Montpellier (CNRS/Université de Montpellier) could help develop treatment strategies for a crippling disorder ...
Aug 1, 2016
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According to studies, approximately one out of every 40 individuals in the United States is a carrier of the gene responsible for spinal muscular atrophy (SMA), a neurodegenerative disease that causes muscles to weaken over ...
Jul 25, 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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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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Spinal muscular atrophy (SMA) is a disease that causes progressive degeneration in the nerve cells that control muscles, thereby causing muscle weakness and eventually death. SMA affects approximately 200,000 people in the ...
May 5, 2016
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Neuroscientists have discovered a specific enzyme that plays a critical role in spinal muscular atrophy, and that suppressing this enzyme's activity, could markedly reduce the disease's severity and improve patients' lifestyles.
Feb 18, 2016
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Researchers at UT Southwestern Medical Center have identified synthetic RNA and DNA that reverses the protein deficiency causing Friedreich's ataxia, a neurological disease for which there is currently no cure.
Feb 16, 2016
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Having a child with a severe genetic condition will heavily impact on a parents' decision to have future prenatal testing, according to a new study co-conducted by Plymouth University.
Jan 20, 2016
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World-leading research from the University of Aberdeen has made a significant breakthrough in understanding a form of childhood motor neuron disease.
Nov 9, 2015
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