This is an observational study. There are no protocol-defined visits, although patients are expected to have routine office visits approximately every 6 months. Upon signing of informed consent/assent and study enrollment, historical data will be abstracted from the patient's medical chart. The patient will then be observed prospectively for up to 5 years, with additional data collected from routine healthcare encounters and direct-to-patient questionnaires (where local laws allow), including laboratory tests, physical exam and patient reported outcomes/quality of life measures. Patients will be consented/assented to provide blood samples for biomarker assessments, DNA sequencing and archiving.
Study Type
OBSERVATIONAL
Enrollment
8
Dokuz Eylul Universitesi Tip Fakultesi
Balçova, Turkey (Türkiye)
Ege University School of Medicine of Pediatric Endocrinology
Bornova, Turkey (Türkiye)
Pediatric Endocrinology and Diabetes Marmara University Hospital
Istanbul, Turkey (Türkiye)
Demographics
Descriptive summary of baseline characteristics including age, sex, ethnicity, and race.
Time frame: Baseline
Medical history
Descriptive summary of medical history over time.
Time frame: 5 years
Clinical course
Descriptive summary of disease progression over time.
Time frame: 5 years
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