New therapies for muscular dystrophy, including cell-based therapies and enzyme inhibitors, are being developed to treat both ambulatory and non-ambulatory forms of the condition. Current treatments for Duchenne muscular dystrophy are limited to corticosteroid agents, but new drugs like givinostat show promise in improving muscle regeneration. Clinical trials are ongoing for various novel therapeutics, including cell-based therapies like HOPE-Duchenne and HOPE-2, as well as enzyme inhibitors for other forms of muscular dystrophy. Combining treatments with different mechanisms of action may provide incremental benefits in treating these rare conditions. The approval process for new treatments may involve an arduous insurance authorization process.
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Cell-based therapies, enzyme inhibitors at vanguard of muscular dystrophy treatment
