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Cystinosis Gene Therapy & Research Updates: Virtual Meetup

Learn more about gene therapy as it relates to the cystinosis community. Dr. Paul Grimm from from Stanford University...

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Open Registration – 2020 Cystinosis Virtual Meetups

We are going virtual! The CRN is introducing a series of virtual meetups taking place this fall. Available 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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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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First Patient Dosed in AVROBIO Phase 1/2 Trial of Gene Therapy for Cystinosis

AVROBIO has announced the first patient has been dosed in the company’s Phase 1/2 trial of AVR-RD-04, an investigational...

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