FAQ

Ultra-rare disorder global patient registries centralize clinical information from a single location allowing researchers to connect with patients. No matter where you live in the world, by joining the Superficial Siderosis Patient Registry, you will have a hand in creating a global portrait of how superficial siderosis presents and progresses. This improved understanding allows physicians to properly diagnose patients and researchers to develop superficial siderosis treatments and the associated symptoms.

All persons 18 years of age or older who have received a diagnosis of superficial siderosis may register. Minors under 18 may have a parent or legal guardian submit their information on their behalf. For adults needing registration assistance, an authorized caregiver or family member may register and submit updates on their behalf.

Rest assured that the Superficial Siderosis Patient Registry takes the utmost care to ensure the confidentiality of your personal information. When you register, you will be asked for some personally-identifying details, but be assured that this information is kept strictly confidential. The only person who can access the identifiable information database is the patient registry administrator, and their access is solely for the purpose of sorting information requests based on criteria like gender, age, or country.

In order to maintain privacy, the questionnaires or surveys you complete may request identifiable information. However, your responses are never stored alongside your name. We employ Research Electronic Data Capture (REDCap), a highly secure web-based application developed by Vanderbilt University for capturing clinical research data and designing projects. This system fully complies with the Health Insurance Portability and Accountability Act (HIPAA).

REDCap automatically removes 18 pieces of identifying information from the data you provide and assigns an anonymous ID number to ensure anonymity. This ID number is linked to a file containing your identifiable information (such as name, address, age, etc.) and is also associated with your responses in the questionnaires. However, your identifiable information is stored separately from your responses, which are tagged only with the ID number, in separate databases.

Your privacy and the security of your personal information are of utmost importance to us, and we have taken rigorous measures to safeguard it within the Superficial Siderosis Patient Registry.

A natural history study aims to track superficial siderosis over time, starting from symptom information prior to diagnosis and continuing until the disorder’s natural progression concludes. This type of study provides a comprehensive understanding of the disorder and identifies the variables that are associated with the patient population and the natural course of the condition. Natural history studies serve various purposes, including the development of best practices for patient care and recruitment for clinical trials.

Clinical trials involve participants who undergo specific interventions according to investigators’ research plan or protocol. These interventions can range from medications, non-prescription dietary supplements, medical devices, and procedures to lifestyle changes. In some cases, clinical trials compare a new treatment approach with the currently available standard therapy, often utilizing a placebo that does not contain active ingredients.

In observational studies, researchers observe patients and collect data without intervening in their diagnostic tests, treatments, or drug interventions. Patients under their physicians’ care provide reports on the effects and outcomes of their treatment plans. Observational studies play a crucial role in gathering real-world data and evaluating the effectiveness of different care approaches.