Clinical trials: Phosphomannomutase II Deficiency
Pmm2-CDG, Phosphomannomutase 2 Deficiency
Oral Epalrestat Therapy in Pediatric Subjects With PMM2-CDG
This is a prospective, single-center, randomized, double-blind, placebo-controlled study designed to assess the safety, tolerability, and clinical and metabolic improvement of pediatric subjects with PMM2-CDG on oral epalrestat…
- Conditions
- Pmm2-CDG, Phosphomannomutase 2 Deficiency, Phosphomannomutase 2 Congenital Disorder of Glycosylation, Phosphomannomutase II Congenital Disorder of Glycosylation
- Trial name
- NCT04925960
- Testing
- Epalrestat
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Maggie's Pearl, LLC