The purpose of the study is to characterize the clinical course of homocystinuria in pediatric and adult patients aged 1 to 65 years under current clinical management practices
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Changes in Met cycle metabolites levels - tHcy
Timeframe: 6.5 years
Changes in Met cycle metabolites levels - total Cys
Timeframe: 6.5 years
Changes in Met cycle metabolites levels - Met
Timeframe: 6.5 years
Changes in Met cycle metabolites levels - Cth
Timeframe: 6.5 years
Patient Reported Outcome (PRO): Quality of Life in Neurological Disorders [Neuro-QoL]
Timeframe: 6.5 years
Patient Reported Outcome (PRO): Quality of Life by 36-Item Short Form Survey [SF-36]
Timeframe: 6.5 years
National Institutes of Health (NIH) Toolbox Cognition Battery
Timeframe: 6.5 years
EuroQol EQ-5D™ questionnaire to measure health and quality-of-life
Timeframe: 6.5 years
Dual-Energy X-Ray Absorptiometry to measure bone mineral density
Timeframe: 6.5 years
Eye assessments to evaluate ocular health: Visual acuity examination will be performed to determine the clarity or sharpness of vision
Timeframe: 6.5 years
Eye assessments to evaluate ocular health: Slit lamp eye examination will be performed to look for any diseases or abnormalities in the anterior portion of the eye
Timeframe: 6.5 years