The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched
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The Brighter Days Care Package Program for Cystinosis Patients and Families has Launched

The Cystinosis Research Network (CRN) has created a new program to bring light to cystinosis families during these uncertain times. It is called the Brighter Days Care Package Program. Brighter…

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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis
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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…

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Study Examines Neurological Differences in Cystinosis Patients
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Study Examines Neurological Differences in Cystinosis Patients

In a study published recently in the Orphanet Journal of Rare Diseases, a team of researchers identified distinct variations in the neuroanatomical and neuropsychiatric phenotypes of cystinosis patients. Phenotype refers to visible,…

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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis
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First Patient Dosed in Phase I/II Study of Experimental Gene Therapy for Cystinosis

According to a recent press release from the Massachusetts-based biotechnology company Avrobio, Inc., the first patient has been dosed in the Company's phase I/II clinical study of their investigational cystinosis…

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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients
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CIRM Awards $12 Million Grant for Clinical Study of Gene Therapy for Cystinosis Patients

According to a press release from the California Institute for Regenerative Medicine (CIRM), the Institute's governing body has approved a grant of nearly $12 million to University of California, San…

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Diagnostic Timeline for Lysosomal Storage Disorders like Fabry Disease Significantly Reduced in South Africa

The Problem Lysosomal storage disorders (LSDs) affect approximately one out of every 7,000 people. There are around 50 different types of LSDs. These include Fabry disease, Batten disease, Gaucher disease,…

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