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4 superficial siderosis research committee summer meeting highlights

4 Superficial Siderosis Research Committee Summer Meeting Highlights


The Superficial Siderosis Research Committee, comprising superficial siderosis experts and patient representatives, recently convened for their summer meeting on June 23, 2023.

Multi-National Deferiprone Double-Blind Clinical Trial 

One of the primary focuses of the meeting was the ongoing planning of a multi-national, double-blind clinical trial using deferiprone, a chelating agent aimed at removing excess iron from the body. Committee Chair Dr. Michael Levy and Professor David Werring provided valuable insights and suggestions to improve the working protocol. Rori Daniel, the SSRA Research Coordinator, will incorporate these edits for further review. Once approved, the protocol will undergo evaluation by the statistical department at Massachusetts General Hospital (MGH), marking a significant step toward establishing a standardized chelation treatment approach for Superficial Siderosis.

Standardized Deferiprone Intervention Guideline 

In addition to the clinical trial, the committee also discussed the development of a standardized deferiprone intervention prescription guideline for physicians. With their expertise, Dr. Levy and Professor Werring will review the patient deferiprone guideline and collaborate on creating a comprehensive physician guideline. This initiative aims to ensure that healthcare professionals worldwide have a clear and consistent approach when administering deferiprone treatment to Superficial Siderosis patients, ultimately improving patient care and outcomes.

Iron Quantification Through Imaging

Accurate quantification of iron deposits in the brain is crucial for monitoring disease progression and evaluating treatment effectiveness. During the email follow-up, Rori Daniel shared her plan to develop a plugin for the OsiriX software, which would automate the Kessler-Levy method for iron quantification. Once completed, this imaging study feasibility assessment will be conducted, offering valuable insights into the disease and potentially paving the way for improved diagnostic techniques and treatment monitoring.

Rhys Holmes shared an update on a Superficial Siderosis Patient Focus Group meeting he recently attended in London in which iron quantification through imaging was discussed.

Redesigning the SSRA-Sponsored Antioxidant Observational Study 

The committee also discussed the redesign of the SSRA-sponsored antioxidant observational study. The plan will transform it into a community-based format conducted through the Superficial Siderosis Patient Registry. This shift aims to enhance patient participation and engagement in research, enabling a more comprehensive understanding of the effects of antioxidants in managing Superficial Siderosis. It will also allow the SSRA to fund the study with donations forgoing the need for a grant application. Professor Werring provided suggestions for potential principal investigators to lead this community-focused study, underscoring the collaborative nature of the research.

In Summary

The Superficial Siderosis Research Committee’s summer meeting marked significant progress, highlighting the commitment and dedication of experts, patients, and researchers to advance our understanding and treatment of Superficial Siderosis. The ongoing efforts to develop a multi-national clinical trial, standardize treatment protocols, quantify iron deposits through imaging, and engage the patient community in research demonstrate a promising future for those affected by this rare disorder. The committee will reconvene in October 2023, continuing their invaluable work towards improving the lives of Superficial Siderosis patients worldwide.

Stay tuned for further updates and developments in this exciting journey of scientific discovery.

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