TRND Projects
NCATS selects TRND projects based on their potential to move forward into human trials and transform patient care for rare and neglected diseases. Each project has the potential for success and ability to teach important lessons about rare and neglected disease drug development. Click on the links below to view current TRND projects:
- Aes-103 for Sickle Cell Disease
- Auranofin for Chronic Lymphocytic Leukemia
- AVI-4038 for Treatment of Duchenne Muscular Dystrophy
- Bone Morphogenetic Protein Inhibitors to Treat Fibrodysplasia Ossificans Progressiva
- CincY as a Treatment for Creatine Transporter Defect
- CMX001 for Treatment of Neonatal Herpes Simplex Virus
- Cyclodextrin for Niemann-Pick Type C1 Disease
- Deuterated Analogs of Praziquantel for Treatment of Schistosomiasis
- Development of the Novel Antifungal VT-1129 for Cryptococcal Meningitis
- Development of Oxadiazoles as Treatment for Schistosomiasis and Hookworm
- Development of VBP15 for Treatment of Duchenne Muscular Dystrophy
- DEX-M74 for Hereditary Inclusion Body Myopathy
- Inhaled GM-CSF Therapy of Autoimmune Pulmonary Alveolar Proteinosis
- A Novel Compound for Targeted Treatment of Core Binding Factor Leukemia
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