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.
News
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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.
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Parents and patients have mixed opinions on preventative bisphosphonates for DMD
Boys with DMD, their parents and health professionals have a mix of perspectives on the use of bisphosphonates to prevent osteoporosis.
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The changing landscape of exon-skipping therapies for DMD
Pharmaceutical companies provided updates on their exon skipping therapies for DMD at the CureDuchenne 2025 FUTURES National Conference.
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Everyday champions: DMD parents and caregivers share stories of connection
A panel at the the CureDuchenne 2025 FUTURES National Conference shared insights from eight families impacted by DMD.
