Global Patient Survey and Collaboration Program for Shwachman-Diamond Syndrome and Related Conditions

What this Trial Is About

The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program SDS-GPS provides a secure online platform for patients and families worldwide to share their experiences living with Shwachman-Diamond Syndrome SDS and related conditions. The program aims to deepen understanding of SDS, enhance patient lives, and speed up the development of new therapies and cures by gathering patient-reported data. SDS-GPS was created with input from patients, families, advocates, researchers, and clinicians to build a supportive global community that drives research forward. Participants create a free account to enter their information and complete surveys covering topics such as demographics, medical history, treatments, disease progression, care access, and quality of life. These surveys can be completed anytime from home and saved for later. The platform supports multiple languages and allows participants to track their medical appointments, medications, and symptoms. The program collects de-identified data to prioritize research, share knowledge, and connect participants with relevant clinical trials and registries. Throughout the program, participants provide ongoing information about symptoms, genetics, quality of life, and disease burden at baseline and at regular intervals such as every 12 months or multiple times per year. This long-term observational approach helps researchers understand patient needs and outcomes. Participation involves no clinic visits or virtual appointments, and privacy and security are maintained at the highest standards. The program offers early access to new research opportunities and supports participants in sharing information with their care teams as they choose.

Shwachman-Diamond Syndrome Global Patient Survey and Partnering Platform