Panelists discuss how hypophosphatemia can cause both acute effects like muscle weakness and confusion and chronic complications such as osteomalacia and metabolic disturbances, potentially impacting patients’ mobility, energy levels, and overall quality of life if not properly monitored and managed.
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EMBARK Data Show Continued Improvements With DMD Gene Therapy
March 19th 2025Data from the EMBARK trial of delandistrogene moxeparvovec in patients with Duchenne muscular dystrophy (DMD) show that benefits in functional outcomes, gene expression, and muscle imaging persist 2 years after receiving the gene therapy.
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March 17th 2025Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach adulthood thanks to transformative therapy advances.
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