Cystinosis symposium: a rare disease model for comprehensive care

TL;DR

A symposium on cystinosis aimed to improve care for patients by fostering collaboration among researchers, healthcare providers, and families.

Contribution

The symposium provided a collaborative platform to address care deficiencies and promote a path toward a cure for cystinosis.

Findings

The symposium highlighted the need for better care strategies for cystinosis patients.

It emphasized the importance of collaboration across healthcare, education, and families.

An algorithm was proposed to guide future efforts toward a cure.

Abstract

The time to provide a forum for collaboration in cystinosis research underscored the recent Cystinosis Symposium: A Rare Disease Model for Comprehensive Care held at the New York Academy of Medicine, on 31 May 2024, an event sponsored by the Cystinosis Research Network and numerous corporate entities (Amgen and others). This conference was one of the first to address the glaring deficiencies in the care of children and adults with cystinosis by focusing on themes that are mutually applicable to multidisciplinary healthcare providers, educators, and families. The ultimate goal was to disseminate new information and awareness to all engaged in the cystinosis community and provide an algorithm to navigate more effectively the promising future toward a cure.