Life Sciences & Medicine Active Updated Aug 11, 2026
Treating Duchenne muscular dystrophy before symptoms appear
Duchenne muscular dystrophy damages muscle from birth even though diagnosis usually comes years later, after standard treatment already starts late. Researchers are now testing whether giving an already-approved drug to toddlers, before scar tissue replaces muscle, can preserve far more function than starting treatment at the usual age.
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Aug 11, 2026 Latest
Binghamton University researchers Eric Hoffman and Raju Nagaraju gave 20 steroid-naive boys aged 2 to under 4 with Duchenne muscular dystrophy daily doses of 2 or 6 milligrams per kilogram of Vamorolone, an already FDA-approved DMD drug, for 12 weeks and reported surprisingly rapid gross motor skill improvements, with most participants continuing treatment for roughly two years through an expanded-access program.
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