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Completed trials with posted results — search by concept, filter by the parameters that matter at the bedside.

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65,437 trials

Phase 3 N=89 Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

Hereditary Inclusion Body Myopathy · Distal Myopathy With Rimmed Vacuoles · Distal Myopathy, Nonaka Type
Primary: Change From Baseline in UEC Score (Total Force in kg) at Week 48 — -2.25; -2.99 kg — p=0.5387
Ultragenyx Pharmaceutical Inc Results Jul 2018 3.4% serious AE View details