The Shwachman-Diamond Syndrome Global Patient Survey and Collaboration Program (SDS-GPS) is an opportunity for patients and their families - from anywhere in the world - to share their experience living with SDS via a safe, secure, and convenient online platform, to * expand the understanding of SDS * improve the lives of people with SDS, and * accelerate the development of new therapies and cures for SDS. By joining, participants will receive early access to relevant information about new clinical trials and other research opportunities (such as clinical registries) based on their profile, accelerating research and increasing clinical trial impact and recruitment success. The platform, consent forms, and surveys are available in five languages: English, Spanish, French, German, and Italian. More languages to come.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Patient (or caregiver) reported symptoms over time
Timeframe: At baseline and every 12 months, prospectively.
Genetics report uploaded by patient (or caregiver)
Timeframe: Through study completion when the genetics report is available.
Quality of life measures via PROMIS surveys
Timeframe: Through study completion, an average of 2-4 times per year.
Patient reported burden of disease and treatment outcomes
Timeframe: At baseline and every 12 months, prospectively.