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Genterapi SPK-8011 forhindrer blødning opptil 4 år

 

Fase 1/2 prøvesett for å teste ASC618, genterapi for hemofili A.

 

EMA OKs Biomarins nye søknad om å godkjenne Roctavian

 

BioMarin sender inn Roctavian gentherapy for EU-godkjenning

 

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Støttet av utdanningsstipend fra Bayer, BioMarin, CSL Behring, Freeline Therapeutics Limited, Pfizer Inc., Spark Therapeutics og uniQure, Inc.

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