Duchenne muscular dystrophy

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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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PTC pulls file for Duchenne therapy on FDA feedback

PTC Therapeutics has abandoned its latest attempt to secure FDA approval for Duchenne muscular dystrophy treatment Translarna.

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Sarepta rises after FDA adds warning label to Elevidys

Uncertainty ends Sarepta's Duchenne gene therapy Elevidys ends after an FDA label update, with the company's shares on the up despite restrictions.

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Sarepta shares slide as DMD treatment woes pile up

The ESSENCE trial of Sarepta's Vyondys 53 and Amondys 45 failed to hit its objective, placing their FDA accelerated approvals at risk.

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Rumour mill says Novartis may want to buy Avidity

Novartis has made an offer to acquire RNA therapeutic specialist Avidity Biosciences, according to an unconfirmed report.

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FDA gives the go-ahead to restart Elevidys shipments

Sarepta has restarted distribution of its Duchenne muscular dystrophy gene therapy Elevidys, but only for patients who can walk.

Regenxbio, Solid report data with Duchenne gene therapies

PTC pulls file for Duchenne therapy on FDA feedback

Sarepta rises after FDA adds warning label to Elevidys

Sarepta shares slide as DMD treatment woes pile up

Rumour mill says Novartis may want to buy Avidity

FDA gives the go-ahead to restart Elevidys shipments

Novartis snaps up PI3Kα breast cancer drug in $3bn deal

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Duchenne muscular dystrophy

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