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Itinai.com biomedical laboratory close up still scene close u e4996bf4 1113 41b3 8fdd 0d1e6c918068 0

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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