Author: cystinosisrn

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Preparing for 2025 – Practical Tips for the New Year 

With the hustle and bustle of the holidays, the new year can certainly sneak up on us. Start 2025 off...

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Novartis Community Statement – Oct 2024

Novartis, the company now leading the investigational stem cell gene therapy trial for cystinosis, has shared a new community...

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Cystinosis Community Takes on Cincinnati, OH

Join us as we celebrate the 7th Annual Cystinosis Awareness Day with an electrifying regional meetup, courtesy of the...

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Cystinosis Symposium Announced for Healthcare Professionals

The New York Academy of Medicine is the location for this “first of its kind” event for healthcare professionals...

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Roslyn Mannon awarded AAKP Medal of Excellence

On the heels of #WorldKidneyDay we congratulate Dr Roz Mannon on her Medal of Excellence. This is one of...

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Eye Care Resource for Cystinosis

Recordati Rare Diseases announced the launch of a new educational website: www.cystinosisinfocus.com. The site aims to help families manage...

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Speak Up, Speak Out 2024

Amgen has partnered with Believe Limited and the Cystinosis Research Network to bring back the third annual, Speak Up,...

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Amgen Acquisition of Horizon Therapeutics Complete

As of October 6, 2023, Amgen officially acquired Horizon Therapeutics. For our cystinosis community using PROCYSBI as a therapeutic...

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Latest Edition Now Online: Cystinosis Advocate Newsletter

The long awaited Cystinosis Advocate Newsletter is here. Choose from research updates, advocacy efforts, family stories, events, cystinosis support...

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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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