← All indications
alpha-Mannosidosis
An inborn error of metabolism marked by a defect in the lysosomal isoform of ALPHA-MANNOSIDASE activity that results in lysosomal accumulation of mannose-rich intermediate metabolites. Virtually all patients have psychomotor retardation, facial coarsening, and some degree of dysostosis multiplex. It is thought to be an autosomal recessive disorder.
Drugs by status
Approved for this indication (1)
Phase 2 trials (10)
Phase 1 trials (1)
Organizations
Organization Involved with Phase 3 Indications (5)
Organization Involved with Phase 2 Indications (4)
Organization Involved with Phase 1 Indications (1)