Clinical trials: GNE Related Diseases
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)
Thrombocytopenia
Efficacy of Sialic Acid GNE Related Thrombocytopenia
Sialic Acid-Extended Release (SA-ER, aceneuramic acid, UX001) is an extended release formulation of sialic acid (SA, also known as N-acetylneuraminic acid or NANA).
- Trial name
- SA-thrombo (NCT02845609)
- Testing
- Sialic Acid-Extended Release
- Volunteers
- 5 people
- Sponsor
- Hadassah Medical Organization