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AVROBIO: Cystinosis Gene Therapy Update Functional & Clinical Improvements Reported

November 17, 2020 – AVROBIO announced that data suggests functional and clinical improvements for the first cystinosis patient at...

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Program to Advance Cystinosis Treatment Options

Partnering with RDMD, this program aims to accelerate cystinosis research to ultimately improve treatment options. We welcome you to...

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Second Patient Dosed in Gene Therapy Trial

AVROBIO announces the second patient with cystinosis has been dosed in the company’s AVR-RD-04 investigational gene therapy Phase 1/2...

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Update: AVROBIO & COVID-19

A letter from Fernanda Copeland, Executive Director, Head Patient Advocacy & Engagement at AVROBIO March 24, 2020 We hope...

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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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