Researchers at Nationwide Children's Hospital have developed a way to measure upper extremity movement in patients with muscular dystrophy using interactive video game technology. Their hope is to expand inclusion criteria ...
Feb 6, 2015
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There is now further evidence that a drug that is effective in treating the rare muscle-wasting disease spinal muscular atrophy (SMA) early in life may be associated with improvement in older children, according to a study ...
Apr 24, 2019
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Federal regulators on Monday granted tentative approval to the first drug for muscular dystrophy, following an intense public campaign from patients and doctors who pushed for the largely unproven medication.
Sep 19, 2016
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A Rutgers-led team may have found the key to preventing Duchenne muscular dystrophy (DMD)-related heart disease, the leading cause of death in patients living with the disease.
Jan 14, 2020
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There are numerous severe, difficult-to-treat monogenetic diseases that are caused by a defect in a single gene—also in the liver. These include the blood coagulation disorders hemophilia A or B or the metabolic disease ...
Oct 26, 2022
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The University of Minnesota Medical School continues its legacy of advancing cell replacement therapies with a scientific breakthrough that highlights the promise of cell therapies for muscular dystrophy.
Feb 14, 2019
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Patients with a cardiac irregularity and myotonic dystrophy type 1 (a severe neuromuscular disorder with a high risk of sudden death) who received an invasive treatment strategy that included testing of their heart's electrical ...
Mar 27, 2012
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Results from a clinical trial of eteplirsen, a drug designed to treat Duchenne muscular dystrophy, suggest that the therapy allows participants to walk farther than people treated with placebo and dramatically increases production ...
Aug 8, 2013
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Scientists have confirmed that mutations of a gene are responsible for some cases of a rare, inherited disease that causes progressive muscle degeneration and weakness: spinal muscular atrophy with lower extremity predominance, ...
May 10, 2012
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Melbourne scientists have shown for the first time how a protein called SMCHD1 helps to police healthy development in the body.
Aug 21, 2018
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