News

EDG-5506 Safely Lowering Muscle Damage Markers in Becker MD Trial

The experimental oral therapy EDG-5506 has been generally well-tolerated and lowered markers of muscle damage among men with Becker muscular dystrophy (BMD) in the open-label ARCH clinical trial, early data show. Findings supported the shift to a higher daily treatment dose in study patients. “These interim 2-month ARCH data…

Defeat Duchenne Canada Grants $1M to DMD Treatment Research

Defeat Duchenne Canada is investing $1.14 million in three research projects aimed at providing better treatments for Duchenne muscular dystrophy, the most common type of muscular dystrophy and one that affects about 1 in 3,500 male births. The trio of three-year projects joins six other research efforts…

Diuretics, BNP Levels Predict Survival in Late-stage DMD: Study

Reduced heart function is associated with a higher rate of mortality among people with Duchenne muscular dystrophy (DMD), a new study reports. Results, however, did not suggest that reduced heart function is an independent predictor of survival for DMD patients. Instead, analyses indicated patients who took diuretics or who…

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…