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Novartis Statement: Cystinosis Stem Cell Gene Therapy Program

In May 2023, Novartis acquired the investigational autologous hematopoietic stem cell (HSC) gene therapy program for the treatment of...

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William A. Gahl, MD, PhD to receive the 2023 Roscoe O. Brady Award

Dr. Gahl is the Director of the Undiagnosed Diseases Program, a Senior Investigator in the Medical Genetics Branch and...

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In Memoriam: Dr. Jerry Schneider

This past winter we lost a revolutionary in the cystinosis world, Dr. Jerry Schneider. During his time at the...

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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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Q & A on Cystaran Eye Drops

Leadiant, the makers of Cystaran eye drops, are opening up the floor for a Town Hall Q&A on Wednesday,...

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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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Update: Cystaran Eye Drop Shortage

As of today, November 12th, AllianceRx Walgreens Prime is unable to fulfill any orders of CYSTARAN. Leadiant’s full announcement...

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CYSTARAN Eye Drop Shortage Anticipated

In a statement from Leadient Biosciences on October 9th, the company announced an availability shortage may impact cystinosis patients...

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Recordati Announces Availability of CYSTADROPS in the U.S.

Beginning September 15, 2020, Recordati Rare Diseases announced the availability of CYSTADROPS across the United States. CYSTADROPS are a...

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Announcement from Leadient

To address the shortage of Cystaran eye drops some members of our community may have encountered recently, Leadient Biosciences...

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