In a first-of-its-kind study, researchers compared the efficacy of preventative therapy for spinal muscular atrophy (SMA) between two well-matched study groups, using either gene therapy (onasemnogene abeparvovec) alone or ...
Jun 25, 2024
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The U.S. Food and Drug Administration has expanded the approval of Sarepta Therapeutics' Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in individuals ≥4 ...
Jun 25, 2024
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The U.S. Food and Drug Administration has approved the first generic version of Emflaza (deflazacort) oral suspension for Duchenne muscular dystrophy (DMD). Approval of the generic version of Emflaza oral suspension was granted ...
Jun 17, 2024
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Researchers from Bar-Ilan University, in collaboration with Sheba Medical Center, have developed a novel platform to model human muscle diseases in the C. elegans worm. This innovation facilitates the study of diseases in ...
May 15, 2024
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Experts are working on novel immune-enhancing therapies called host-directed therapies to use the body's own immune system to target tuberculosis, with hopes that they could tackle even the drug-resistant forms of the disease. ...
Apr 26, 2024
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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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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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