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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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Rare Disease Day 2022

February 28th marks the 12th annual Rare Disease Day (RDD). Observed on the rarest day of the year, the...

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Research Update: Investigational Gene Therapy for Cystinosis

Data from the AVROBIO Phase 1/2 investigational gene therapy for cystinosis was released today at WORLDSymposium in San Diego....

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ALAB Podcast Release: Coping Mechanisms

The Adult Leadership Advisory Board (ALAB) continues their podcast series “Cystinosis Rare: Journey Into the Unknown” with episode 8...

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Cystinosis Case Competition

AdvoSci Health hosted a Cystinosis Case Competition this fall semester. Students were tasked with learning foundational information about cystinosis...

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How A Rare Diagnosis Impacts A Family

The Dicks Family sat down for a candid conversation with Daniel Levine from Global Genes. During the latest podcast,...

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Over $40k in Cystinosis Scholarships Awarded

This summer we reached a milestone – over $40,000 in scholarships awarded since our program’s inception. Congratulations to the...

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May 2021 Cystaran Update

Please click here for the latest updates from Leadiant on the Cystadrops shortage. If you have further questions, please...

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An Open Letter to the Cystinosis Community

Interim CRN President, Tim Wyman, has written an open letter to the cystinosis community. You can read it here.

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Rare Disease Day 2021

The goal of Rare Disease Day is to raise awareness amongst the general public and decision-makers about rare diseases and their impact on...

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