Duchenne muscular dystrophy

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FDA advisors vote no to Capricor's DMD therapy

FDA advisors voted that Capricor has not shown efficacy for deramiocel in Duchenne muscular dystrophy, due to altered endpoints in its main trial.

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Servier wagers $2.65bn on Edgewise muscular dystrophy drug

Servier has signed the largest licensing deal in its history, paying $1.55bn upfront for Edgewise's muscular dystrophy business headed by sevasemten.

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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.

FDA advisors vote no to Capricor's DMD therapy

Servier wagers $2.65bn on Edgewise muscular dystrophy drug

Regenxbio's Duchenne gene therapy clears phase 3 hurdle

Deal finally unlocks NHS access to ITF's Duchenne drug

Capricor sues partner Nippon Shinyaku in DMD therapy spat

Regenxbio, Solid report data with Duchenne gene therapies

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Duchenne muscular dystrophy

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