Findings from a recent study in the Journal of Neuromuscular Diseases demonstrate the effectiveness of disease-modifying treatments (DMTs) in infants with spinal muscular atrophy (SMA). The study's results add further support ...
Apr 15, 2024
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The lab of Yongchao C. Ma, Ph.D., at Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago has discovered a fundamental biological mechanism that could lead to new treatments ...
Apr 5, 2024
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The U.S. Food and Drug Administration has approved Duvyzat (givinostat) as an oral medication for the treatment of Duchenne muscular dystrophy (DMD) in patients six years of age and older.
Mar 26, 2024
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For the thousands of people globally affected by the disease myasthenia gravis (MG), everyday activities become a struggle, and in severe cases, the disease can be life-threatening.
Mar 21, 2024
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Ravindra Singh has spent years studying a gene that, when missing or mutated, causes spinal muscular atrophy (SMA), a deadly disease that's among the most common genetic disorders in children. His team's latest work will ...
Mar 13, 2024
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Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a ...
Mar 6, 2024
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A specific gene may play a key role in new treatments that prevent muscle in the body from breaking down in serious muscle diseases, or muscular dystrophies. This is shown in a new study carried out at Umeå University, Sweden, ...
Mar 6, 2024
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The drug vamorolone (Agamree) has been hailed as a promising new drug to treat Duchenne muscular dystrophy (DMD). It has recently been approved for clinical use in the United States by the Food and Drug Administration (FDA) ...
Jan 25, 2024
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Muscular dystrophies are a group of diseases in which proteins in the muscles do not function properly, either through inherited or spontaneous mutations. This, in turn, leads to muscle tissue breaking down over time and ...
Jan 22, 2024
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About 100 cancer predisposition genes (CPGs) are known. The probability of developing cancer is greater in individuals who inherit certain altered variants of one of these genes.
Jan 16, 2024
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