Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

NCT02736188 · clinicaltrials.gov ↗

PHASE3

Phase

TERMINATED

Status

143

Enrollment

INDUSTRY

Sponsor class

Conditions

Hereditary Inclusion Body Myopathy
Distal Myopathy With Rimmed Vacuoles
Distal Myopathy, Nonaka Type
GNE Myopathy
Quadriceps Sparing Myopathy

Interventions

DRUG: Aceneuramic Acid Extended-Release Tablets

Sponsor

Ultragenyx Pharmaceutical Inc