genetic diseases
Acadia closer to bringing first Rett syndrome drug to market
Phil Taylor
Acadia Pharmaceuticals, genetic diseases, Neuren Pharmaceuticals, rare disease, Rett Syndrome, trofinetide
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Acadia closer to bringing first Rett syndrome drug to market
https://pharmaphorum.com/news/acadia-closer-to-bringing-first-rett-syndrome-drug-to-market/
Catabasis craters after giving up on muscular dystrophy lead
Phil Taylor
Catabasis, Duchenne muscular dystrophy, edasalonexent, genetic diseases
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Catabasis craters after giving up on muscular dystrophy lead
https://pharmaphorum.com/news/catabasis-craters-after-giving-up-on-muscular-dystrophy-lead/
EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD
Phil Taylor
bluebird bio, ema, Europe, gene therapy, genetic diseases, rare disease
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EMA starts rapid review of Bluebird’s gene therapy for rare disease CALD
https://pharmaphorum.com/news/ema-starts-rapid-review-of-bluebirds-gene-therapy-for-rare-disease-cald/