Development of an Infantile GM2 Clinical Rating Scale: Remote Assessment of Clinically Meaningful Health-Related Function

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Practical Solution for GM2 Gangliosidoses

Challenges Addressed:

• Rare lysosomal storage disorder causing central nervous system damage
• Lack of feasible clinical outcome assessments for GM2

Value Offered:

• Potential for improved outcomes with early diagnosis and pharmacotherapies
• Development of Infantile GM2 Clinical Rating Scale for health-related function assessment

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