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Results for “Nonaka Disease”

6 trials

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Showing 6 of 6 results

Not applicableStudy completedNCT01784679
What this trial is testing

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Who this might be right for
Hereditary Inclusion Body MyopathyGNE MyopathyNonaka Disease+2 more
Ultragenyx Pharmaceutical Inc 319
Not applicableStudy completedNCT01417533
What this trial is testing

A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases

Who this might be right for
GNE MyopathyGNE Related Diseases
National Human Genome Research Institute (NHGRI) 78
Not applicableUnknownNCT04009226
What this trial is testing

International GNE Myopathy Patient Registry

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Newcastle University 430
Not applicableStudy completedNCT01902940
What this trial is testing

Natural History in CCFDN and IBM Syndromes

Who this might be right for
Inclusion Body Myositis, SporadicInclusion Body Myopathy, Autosomal-recessiveInclusion Body Myopathy, Autosomal-dominant+1 more
Ludwig-Maximilians - University of Munich 350
Testing effectiveness (Phase 2)Study completedNCT01517880
What this trial is testing

Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy

Who this might be right for
GNE MyopathyHereditary Inclusion Body Myopathy
Ultragenyx Pharmaceutical Inc 46
Large-scale testing (Phase 3)Study completedNCT04671472
What this trial is testing

Efficacy Confirmation Study of NPC-09

Who this might be right for
GNE MyopathyDistal Myopathy With Rimmed Vacuoles (DMRV)Hereditary Inclusion Body Myopathy (hIBM)+1 more
Nobelpharma 14