Hereditary Inclusion Body Myopathy-Disease Monitoring (HIBM-DMP): A Combined Registry and Prospective Observational Natural History Study to Assess HIBM Disease
Conditions:   Hereditary Inclusion Body Myopathy;   GNE Myopathy;   Nonaka Disease;   Quadriceps Sparing Myopathy (QSM);   Distal Myopathy With Rimmed Vacuoles (DMRV)Intervention:   Sponsors:   Ultragenyx Pharmaceutical Inc;   TREAT-NMDRecruiting - verified September 2013 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 4, 2013 Category: Research Source Type: clinical trials

Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
Conditions:   Hereditary Inclusion Body Myopathy (HIBM);   GNE MyopathyIntervention:   Drug: ManNAcSponsors:   National Human Genome Research Institute (NHGRI);   National Center for Advancing Translational Science (NCATS);   Therapeutics for Rare and Neglected Diseases (TRND)Completed - verified March 2015 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - July 3, 2012 Category: Research Source Type: clinical trials

Follistatin Gene Transfer to Patients With Becker Muscular Dystrophy and Sporadic Inclusion Body Myositis
Conditions:   Becker Muscular Dystrophy;   Sporadic Inclusion Body MyositisIntervention:   Biological: rAAV1.CMV.huFollistatin344Sponsors:   Nationwide Children's Hospital;   Parent Project Muscular Dystrophy;   The Myositis Association (Grant Sponsor)Enrolling by invitation - verified February 2015 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 23, 2012 Category: Research Source Type: clinical trials

A Natural History Study of Patients With Hereditary Inclusion Body Myopathy
Condition:   Hereditary Inclusion Body MyopathyIntervention:   Sponsors:   National Human Genome Research Institute (NHGRI);   Therapeutics for Rare and Neglected Diseases (TRND)Recruiting - verified April 2014 (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - August 13, 2011 Category: Research Source Type: clinical trials