Click on the links below to view active TRND projects:
- A Novel Compound for Targeted Treatment of Core Binding Factor Leukemia
- A Protein Replacement Drug for Friedreich’s Ataxia
- Bone Morphogenetic Protein Inhibitors to Treat Fibrodysplasia Ossificans Progressiva
- Development of Malaria Transmission-Blocking Drugs
- Gene Therapy for the Treatment of AADC Deficiency
- Gene Therapy for the Treatment of Pompe Disease
- Inhaled GM-CSF Therapy of Autoimmune Pulmonary Alveolar Proteinosis
- Long-Acting Parathyroid Hormone Analog for the Treatment of Hypoparathyroidism
- LUM-001 as a Treatment for Creatine Transporter Deficiency
- Repurposing an EU Therapeutic for Hemoglobinopathies
- Therapy for Fuchs Endothelial Corneal Dystrophy
- Use of Rapamycin for the Treatment of Hypertrophic Cardiomyopathy in Patients with LEOPARD Syndrome
Patient-focused collaboration among government and academic scientists enabled the