News

Review Identifies 23 Factors Affecting Duchenne MD Prognosis

Treatment with glucocorticoids and heart medicines are key factors in determining the prognosis of Duchenne muscular dystrophy (DMD), according to a study. Results also highlighted genetics as an important factor affecting DMD progression. The paper, “Prognostic indicators of disease progression in Duchenne muscular dystrophy: A literature review…

Tamoxifen Increases Survival of DMD Heart Muscle in Cell Study

Treatment with the breast cancer medicine tamoxifen improved the functionality and survival of heart muscle cells in a cellular model of Duchenne muscular dystrophy (DMD), a study reports. The study, “Tamoxifen treatment ameliorates contractile dysfunction of Duchenne muscular dystrophy stem cell-derived cardiomyocytes on bioengineered substrates,” was published…

More Data Support Gene Therapy for DMD Exon Duplications

New data from a Phase 1/2 clinical trial of an exon-skipping gene therapy suggest it may help preserve muscle function if delivered early on to Duchenne muscular dystrophy (DMD) patients with exon duplications. This is the first time a clinical trial has shown production of full-length dystrophin, the protein…

MDA Campaigns Support Summer Camp, NMD Community

To help support its popular summer camp and the neuromuscular disease community, the Muscular Dystrophy Association (MDA) has launched campaigns through October that involve more than 1,000 retailers nationwide. Proceeds from the MDA’s summer retail campaigns will benefit research, care, and advocacy for those living with muscular…