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DMD

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Sarepta Therapeutics’ DMD gene therapy shows early success

Most patients with Duchenne muscular dystrophy require full-time use of a wheelchair by their early teens

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FDA approves Sarepta rival NS Pharma’s Duchenne drug

Drug approved for patients with exon 53 mutation

Roche Basel Switzerland

Roche pays $1.15bn for Sarepta’s Duchenne gene therapy

Pens another gene therapy deal following takeover of Spark Therapeutics

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Sarepta rival NS Pharma files Duchenne drug with FDA

Another blow for Sarepta following FDA rejection earlier this year

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FDA rejects Sarepta’s new Duchenne drug over safety fears

FDA gave no warnings of concerns, says chief exec

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Sarepta investigating mistakenly submitted Duchenne therapy report

Study participant fell seriously ill

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Rivals rise as safety issue mars Pfizer’s DMD gene therapy trial

Company to press ahead with phase 3 trial

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Acer Therapeutics hit by FDA filing rejection

Shares plunge as regulator calls for new trial

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Vertex eyes Duchenne market with Exonics, CRISPR deals

Biotech has gene-editing expertise

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Duchenne UK backs Evox dystrophin delivery project

Funding will drive preclinical studies on dystrophin-bearing exosomes

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Pfizer abandons antibody for Duchenne

Latest to fail in myostatin inhibitor class

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Daily Brief: Sarepta deepens gene therapy investment, NICE changes mind on Besponsa, Digital Medicine company Akili raises $13m

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