Regenxbio's Gene Therapy Helps Kids with Deadly Muscle Disease
Children with a deadly muscle disorder had more control of their bodies after getting an experimental gene therapy from Regenxbio Inc. in a small study. The results foreshadow a potential rivalry with Sarepta Therapeutics Inc. and a critical test for the Trump administration's new drug regulators.
The first five children with Duchenne muscular dystrophy to receive a higher dose of Regenxbio's treatment showed 'consistent benefit' after nine months, with improvements in motor function and the time it took to stand, walk and climb, the company said Thursday. It's conducting a larger study involving about 30 patients that it expects to be in full swing by the end of the year, with the potential to file for approval by mid-2026.
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