Research on key gene networks expands understanding of DMD
Research showed five major genes driving inflammation, immune imbalance and scarring in Duchenne muscular dystrophy.
News
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Growth hormone stimulator shows modest benefits in severe DMD mouse model
A DMD mouse study found some muscle benefits with the compound JMV2894, and shows how disease severity can change treatment response.
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Long-term data indicates vamarolone is as effective as standard steroids, but safer
A press release indicates that vamarolone may be as effective as the standard corticosteroids used for DMD, but with a better safety profile.
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Study highlights impact of body fat, blood pressure on heart health in DMD
Modifiable cardiovascular risk factors are associated with adverse changes in the heart muscle of DMD patients.
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Ruxolitinib extends lifespan and strengthens muscle in Duchenne model
Ruxolitinib reduced harmful senescent cells and improved muscle and bone in a mouse model of Duchenne muscular dystrophy.
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Caregivers report meaningful gains after gene therapy in EMBARK DMD trial
New research emphasizes that achieving even modest functional improvements in DMD is meaningful to families.
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Regular use of lung volume recruitment therapy low in DMD
Fewer than half of participants with DMD who were prescribed LVR therapy adhered to their treatment protocol, a study found.
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Muscle protein desmin may protect the heart in DMD
Treatments that boost levels of the muscle protein desmin may help protect the heart in people with DMD.
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Microdystrophin gene therapy improves muscle function but raises heart risks
A gene therapy called GNT0004 improved muscle and heart function in DMD but caused heart rhythm problems at high doses.
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Trehalose may improve effects of microdystrophin therapy in DMD
Researchers found that trehalose further improved cellular function in animal models of DMD treated with microdystrophin therapy.
