For more details about clinical research studies supported by TRND, click on the links below or visit ClinicalTrials.gov.
Active Studies
Creatine Transporter Deficiency
GNE Myopathy (previously known at Hereditary Inclusion Body Myopathy)
Niemann-Pick Disease Type C
Completed Studies
Chronic Lymphocytic Leukemia
GNE Myopathy (previously known at Hereditary Inclusion Body Myopathy)
- A Phase I Study to Evaluate the Safety and Tolerability of ManNAc in Subjects GNE Myopathy
- A Phase II Study of DEX-M74 (ManNac) in Subjects with GNE Myopathy
Patient-focused collaboration among government and academic scientists enabled the 
Watch the following videos about the rare childhood disease Niemann-Pick type C (NPC):