Patients and families value self-care, muscle and heart function in DMD treatment
Researchers interviewed patients with DMD and caregivers to assess which key treatment characteristics matter most to them.
News
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Satellos reports promising early results for SAT-3247 in DMD
SAT-3247, an investigational oral therapy targeting muscle regeneration in DMD, improved grip strength in a small clinical trial.
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Glucocorticoids may delay respiratory decline in DMD
The use of corticosteroid treatment could delay respiratory failure in patients with Duchenne muscular dystrophy (DMD).
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Experimental drug improves gut-muscle crosstalk in DMD mice
Researchers found that targeting gut microbiome dysfunction in a mouse model of DMD improves muscle health and function.
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New stem cell therapy to be tested in patients with Duchenne muscular dystrophy
A new cell therapy called MyoPAXon has entered early testing in patients with Duchenne muscular dystrophy.
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New research demonstrates lasting benefits of tRNA therapy in DMD models
Early research indicates the therapy could restore nearly 70% of healthy dystrophin protein in people with DMD caused by nonsense mutations.
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Fabric ‘exosuit’ designed to boost arm movement in DMD
A new wearable “soft exosuit” developed by researchers in Korea may help people with DMD lift their arms and shoulders.
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Quantitative muscle ultrasound offers noninvasive method for monitoring DMD
QMUS is a useful tool that correlates with some aspects of a patient’s functional capacity.
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Medicaid spending on DMD drugs soars nearly 3,000% in 5 years
Utilization and costs of novel DMD therapies has risen rapidly in recent years, raising questions about affordability and equitable access
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NS-051/NCNP-04 granted FDA Orphan Drug designation for DMD
Patients with Duchenne muscular dystrophy may benefit from a potential therapy now supported by an FDA orphan designation.
