The objective of this study is to evaluate the natural history of disease progression in infants with globoid cell leukodystrophy (GLD).
This study is being conducted to gather prospective data on disease progression in infants diagnosed with GLD. This study will be performed using protocol-defined, standardized assessments including clinical, developmental, and neurologic measures. All study visits will be conducted in the subject's home. No travel to the study site is necessary.
Study Type
OBSERVATIONAL
To measure the change from baseline in growth parameters (eg, weight gain, linear growth, head circumference).
Time frame: 1 year
To determine the onset date of inadequate oral nutrition, hydration, and/or ventilation as a biomarker for survival
Time frame: 1 year
To assess the change from baseline in clinical parameters of GLD disease progression from a standardized infant neurological examination and infant distress scales.
Time frame: 1 year
To assess the change from baseline in clinical parameters described in Hagberg's clinical staging.
Time frame: 1 year
To measure the time to absolute survival
Time frame: 1 year
To assess the AE experience in this patient population
Time frame: 1 year
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