For more details about clinical research studies supported by TRND, click on the links below or visit ClinicalTrials.gov.
GNE Myopathy (previously known at Hereditary Inclusion Body Myopathy)
- A Phase II Study of DEX-M74 (ManNac) in Subjects with GNE Myopathy
- A Natural History Study of Patients with GNE Myopathy
Niemann-Pick Disease Type C
- Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
- Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C