Glucocorticoid steroids are used to treat a variety of conditions, including chronic inflammatory diseases and autoimmune diseases. Their effects on muscle recovery and repair make them particularly effective therapies for ...
May 8, 2017
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When Marathon Pharmaceuticals announced in February it would market a drug for treating Duchenne muscular dystrophy for US$89,000 a year, the negative reaction was so intense that the company immediately suspended the rollout. ...
Mar 17, 2017
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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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I began writing about genetics decades ago, and the best thing about getting older is witnessing the development of targeted treatments for single-gene diseases that I never thought would happen. But it is happening, for ...
Oct 1, 2015
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In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already ...
Jul 17, 2014
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To make up for insufficient amounts of SMN protein, the cause of the inherited neuromuscular disease spinal muscular atrophy (SMA), researchers have successfully delivered a replacement SMN1 gene directly to the spinal cords ...
May 22, 2014
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NYU biologists have created model neurons with greater precision and efficiency than have been achieved in the past. Their breakthrough, which appeared this fall in a pair of papers in the journal Nature Neuroscience, has ...
Dec 12, 2013
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(AP)—Doctors are preparing an emergency one-off stem cell treatment for 2-year-old Venetian girl suffering a severe muscular disease after a judge revoked an order blocking the cure.
Aug 23, 2012
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