Clinical trials: Hereditary Inclusion Body Myopathy (HIBM)
GNE Myopathy, GNE Related Diseases
A Natural History Study of Patients With GNE Myopathy and GNE-Related Diseases
Background: GNE Myopathy is a disease that causes walking difficulties and increasing muscle weakness. It usually develops in young adults (between 20 and 30 years of age), and affects arm and leg muscles.
- Trial name
- NCT01417533
- Where
- United States
- Volunteers
- 78 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Hereditary Inclusion Body Myopathy, GNE Myopathy
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)
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and…
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM)
- Trial name
- NCT01784679
- Where
- 5 sites in United States, Bulgaria and 3 more
- Volunteers
- 319 people
- Sponsor
- Ultragenyx Pharmaceutical Inc