DMD associated with high household costs that rise as the disease advances
Accommodating the functional impairments associated with DMD appears to involve substantial costs for households.
News
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Capricor Therapeutics announces positive data from study of DMD cell therapy
Deramiocel continues to show effectiveness in treating DMD in a currently ongoing trial.
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Higher cholesterol levels associated with decreased bone density in DMD
Cholesterol and triglyceride levels appear to be inversely correlated with bone density in patients with Duchenne muscular dystrophy (DMD).
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New gene therapy delivery approach restores full-length dystrophin in DMD mice
A new gene therapy using triple AAV vectors restored full-length dystrophin and improved muscle function in mice with DMD.
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Sarepta shares plans to improve safety of Elevidys
Following two fatal cases of acute liver failure associated with ELEVIDYS, Sarepta is taking steps to improve the safety of the drug.
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Children’s Hospital of Philadelphia granted Certified Duchenne Care Center status
Parent Project Muscular Dystrophy has recognized the Children’s Hospital of Philadelphia’s excellence in providing care for patients with DMD.
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REGENXBIO shares positive findings from DMD gene therapy trial
Preliminary results from the AFFINITY DUCHENNE trial show that the gene therapy RGX-202 improves functional performance in patients with DMD.
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Parents need better awareness of respiratory care for DMD, study finds
Although the parents of children with DMD understand the basics of pulmonary problems, they lack knowledge about advanced respiratory care.
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Novel metabolism-targeting drug may improve DMD symptoms
A recent study found that a mouse model of DMD treated with a novel enzyme inhibitor exhibited improvements in muscle function.
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Prenatal stress shapes future health in mouse model of DMD
Stress during late pregnancy changed how Duchenne muscular dystrophy (DMD) impacted offspring in a mouse model.
