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Table 2 MDRI domains, MID thresholds, and study population from the Phase 3 Study of Laronidase

From: The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases

Domain MID threshold Study population with baseline deficiency in domain
6MWT  ± 54 m Approximately 50% < 350 m
FVC  ± 11% FVC < 80% was entry criterion; 100% qualify
Shoulder ROM  ± 20° Approximately 50% had ROM < 90°
AHI  ± 10 events per hour Approximately 50% AHI > 10
Visual acuity  ± 2 lines 8 Patients (approximately 20%) > 20/200
  1. MID minimally important difference, 6MWT six minute walk test. FVC forced vital capacity, ROM range of motion, AHI apnea hypopnea index
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172

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