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  1. Home
  2. About NCATS
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  4. Therapeutics for Rare and Neglected Diseases (TRND)
  5. TRND Projects
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Active TRND Projects

Click on the links below to view active TRND projects:

  • Antifibrotic Therapy for the Treatment of Pulmonary Hypertension
  • Bone Morphogenetic Protein Inhibitors to Treat Fibrodysplasia Ossificans Progressiva
  • Development of Acoziborole for the Treatment of Human African Trypanosomiasis
  • 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 for Autoimmune Pulmonary Alveolar Proteinosis
  • LUM-001 as a Treatment for Creatine Transporter Deficiency
  • A Novel Compound for Targeted Treatment of Core Binding Factor Leukemia
  • Novel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis
  • A Protein Replacement Drug for Friedreich’s Ataxia
  • Repurposing an EU Therapeutic for Hemoglobinopathies
  • Repurposing of a Long-Acting Parathyroid Hormone Analog for the Treatment of Hypoparathyroidism
  • ​Therapy for Fuchs Endothelial Corneal Dystrophy
  • A Treatment for Patients with Jansen’s Metaphyseal Chondrodysplasia
  • Use of Rapamycin for the Treatment of Hypertrophic Cardiomyopathy in Patients with Noonan Syndrome with Multiple Lentigines (NSML)

Contact

TRND Program staff

Gene Therapy Advances to Clinical Trial

TRND Pompe image​NCATS and Duke University scientists help advance a gene therapy for Pompe into clinical testing for the first time.

Last updated: 11-02-2020

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