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.
News
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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.
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Study identifies a critical window for orthopedic surgery in DMD
Orthopedic surgery in patients with DMD should be performed within the first year after initial gait loss for a higher chance of success.
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Transitioning from pediatric to adult care remains a challenge in DMD
A new review finds ongoing gaps in how people with DMD move from pediatric to adult care, and urges better coordination between care teams.
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Blood proteins may predict how quickly DMD will progress
Blood tests could one day help doctors predict how quickly DMD will progress and measure how well new treatments are working.
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Risk tolerance for gene therapy rises with DMD progression, survey finds
A new study assessed the maximum risk of treatment-related death that people would accept to receive gene therapy for DMD.
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Synthetic molecule prevents DMD in mouse model
A study found that a synthetic compound known as a BB polymer may protect skeletal and cardiac muscles from damage in DMD.
