Antigen-Peptide-Transporter 2 Deficiency Overview
Learn About Antigen-Peptide-Transporter 2 Deficiency
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Summary: Owing to the rarity, severity, speed of progression and fatal prognosis of infantile and juvenile GM1, there is a limited understanding of overall disease progression and meaningful outcome measures. This study aims to build a natural history data set through collection of a number of clinical, imaging, and laboratory assessments that may be specific predictors of GM1 disease progression and clini...