Duchenne muscular dystrophy

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Regenxbio's Duchenne gene therapy clears phase 3 hurdle

Regenxbio is pitching at FDA approval in 2027 for its Duchenne muscular dystrophy gene therapy RGX-202, a potential rival to Sarepta/Roche's Elevidys.

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Deal finally unlocks NHS access to ITF's Duchenne drug

After nearly two years of wrangling, ITF's Duvyzat can be used by the NHS to treat people living with Duchenne muscular dystrophy.

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Capricor sues partner Nippon Shinyaku in DMD therapy spat

Capricor has filed a lawsuit seeking to split from its commercial partner for Duchenne muscular dystrophy therapy, deramiocel, in the US.

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Boy in a wheelchair looking out to the forest

Regenxbio, Solid report data with Duchenne gene therapies

Regenxbio and Solid Bio say their gene therapies for Duchenne muscular dystrophy show no sign so far of the liver issues seen with Sarepta's Elevidys.

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