In 2013, Zoe Harting became the first baby in the world to receive an experimental drug that her doctors hoped would save the lives of thousands of infants like her.
Dec 6, 2016
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(Medical Xpress)—A University of Alberta researcher has pinpointed a mutation that brings the medical community another step closer to treating those who suffer from a fatal type of muscular dystrophy.
Jul 30, 2013
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A large scale scientific collaboration led by France's Pierre and Marie Curie University and national institute of health and medical research has generated significant advances in treatment for muscular dystrophies by targeting ...
Oct 25, 2013
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The challenge of treating patients with genetic disorders in which a single mutated gene is simply too large to be replaced using traditional gene therapy techniques may soon be a thing of the past. A Nationwide Children's ...
Jul 9, 2012
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A team of scientists led by Pier Lorenzo Puri, M.D., associate professor at Sanford-Burnham Medical Research Institute (Sanford-Burnham), in collaboration with Fondazione Santa Lucia in Rome, Italy, have published details ...
Apr 14, 2014
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For the first time, scientists found that in spinal muscular atrophy (SMA), the affected nerve cells that control muscle movement, or motor neurons, have defects in their mitochondria, which generate energy used by the cell. ...
Sep 15, 2016
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Results of a compassionate-use study released in Stem Cells Translational Medicine show promising results for treating muscular dystrophies with mesenchymal stem cells (MSCs) derived from Wharton's jelly (WJ), a substance ...
Jul 27, 2021
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Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. The disease often results in death before ...
Aug 27, 2020
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Facioscapulohumeral muscular dystrophy (FSHD) is caused by aberrant expression of the DUX4 gene in skeletal muscles. Researchers at Nationwide Children's Hospital have recently demonstrated that an endogenous human microRNA, ...
Dec 14, 2021
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Researchers at the University of Missouri have identified a communication breakdown between nerves and muscles in mice that may provide new insight into the debilitating and fatal human disease known as spinal muscular atrophy ...
Sep 26, 2011
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