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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 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…
The Muscular Dystrophy Association (MDA) is hosting a one-day games streaming fundraiser, dubbed “MDA Rivals,” this Saturday, June 18. The event, streaming on MDA’s Twitch channel, will feature stars from MDA Let’s Play — a platform that aims to raise funds for MDA and awareness about muscular…
FibroGen has completed patient enrollment for its Phase 3 trial evaluating pamrevlumab in combination with corticosteroids as a potential treatment for children with Duchenne muscular dystrophy (DMD). The LELANTOS-2 study (NCT04632940) has enrolled 73 boys with DMD, ages 6 to 12, who maintain their ability to walk.
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…
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…
Despite broad initiatives seeking improvements, the time it takes an individual with Duchenne muscular dystrophy (DMD) to get a correct diagnosis has remained largely unchanged over the past three decades, a study reports. It still takes more than two years, on average, from the time symptoms are first noticed…
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…
The FSHD Society has joined forces with the Critical Path Institute (C-Path) to facilitate the collection of data from people with facioscapulohumeral muscular dystrophy (FSHD) who were given a placebo in previous clinical trials. Provided by sponsors of FSHD trials, the data will be integrated into C-Path’s…
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…
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