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Brochure Available: Details Gene Therapy Used in Cystinosis Trial

AVROBIO has released a brochure providing detail on investigational gene therapies for lysosomal storage disorders. Earlier this year, AVROBIO’s...

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Invitation: Clinical Trial for Cystinosis Nonsense Mutation

NoMuRiC (Nonsense Mutation Readthrough in Cystinosis) A Phase II Clinical Trial of ELX-02 for Treatment of Cystinosis (McGill University...

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