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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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FDA advisors narrowly back Sarepta’s Duchenne gene therapy

An FDA advisory committee has given tentative backing to Sarepta’s gene therapy for Duchenne muscular dystrophy (DMD), even though the agency’s reviewers said they were no

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2019-Gene-Therapy-Muscular-Dystrophy_ProgFeature_IMG

Solid Biosciences acquires AavantiBio, conducts $75 million ...

The life sciences company Solid Biosciences is merging with the privately-held gene therapy company AavantiBio in a

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Myocarditis case mars Sarepta DMD gene therapy readout

New data on Sarepta's gene therapy for Duchenne muscular dystrophy (DMD) has gone a long way towards building confidence in its efficacy – but have introduced a concern about its safety.

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Young lonely man in a wheelchair on a balcony looking at the nature in the spring

Sarepta says early filing for DMD gene therapy may be back o...

The development of gene therapies for Duchenne muscular dystrophy has proved to be challenging, but one of the key players – Sarepta – thinks it may have the clinical data needed to file fo

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Pfizer pauses Duchenne gene therapy trial after patient deat...

Pfizer has called a halt to a clinical trial of its gene therapy for Duchenne muscular dystrophy as it investigates the unexpected death of a young male patient.

Regenxbio, Solid report data with Duchenne gene therapies

FDA advisors narrowly back Sarepta’s Duchenne gene therapy

Solid Biosciences acquires AavantiBio, conducts $75 million ...

Myocarditis case mars Sarepta DMD gene therapy readout

Sarepta says early filing for DMD gene therapy may be back o...

Pfizer pauses Duchenne gene therapy trial after patient deat...

FDA approves higher Wegovy dose via 4th priority voucher

Using AI-driven synthetic personas to take your insights fur...

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