treatment

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...

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

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

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

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

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

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

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

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

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