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AVROBIO Reports Positive Data From Phase 1/2 Clinical Trial of Investigational Gene Therapy for Cystinosis, Including New Interim Data on Neurocognitive Measures

Press release originally published May 17, 2022 on Avrobio.com. New early data show key visual motor integration, visual perception...

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Newborn Screening: Review of its Impact for Cystinosis

Could newborn screening provide a turning point in the natural history of cystinosis? Review the latest publication “Newborn Screening: Review of...

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Shortage Impacting WBC Testing Kits

An announcement from Horizon Therapeutics: With the growing dry ice shortage there has been an increased demand for NanoCool...

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Survey: Impact of COVID-19 + Rare Diseases

The Rare Diseases Clinical Research Network (RDCRN) is hoping to learn how the COVID-19 pandemic impacts people who live...

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