Rare Disease & Orphan Indications

Rare Disease & Orphan Indications

Rare Disease and Orphan Indication Drug Experience

Medpace has conducted more than 100 global trials covering Phase I-IV including these rare disease indications.

  • Acromegaly
  • Adrenocortical carcinoma (ACC)
  • atypical Hemolytic Uremic Syndrome (aHUS)
  • Cushing’s Syndrome
  • Cystic Fibrosis
  • Duchenne Muscular Dystrophy (DMD)
  • Eosinophilic Esophagitis
  • Familial Hypercholesterolemia
  • Fibrodysplasia Ossificans Progressiva
  • Gastroparesis
  • Glomerulonephritis
  • Growth Hormone Deficiency
  • Lysosomal Storage Diseases
  • Paroxysmal Nocturnal Dyspnoea (PND)
  • Renal vasculitis
  • Respiratory Syncytial Virus (RSV) in lung transplant patients
  • Tinnitus
  • TTR Amyloidosis
  • Retinitis Pigmentosa
  • Usher’s Syndrome

Focused Rare Disease Teams Deliver Results

  • Committed cross-functional team of Rare Disease experts comprised of doctors, project managers, regulatory consultants, and late phase specialists to orchestrate these complex studies
  • Board-certified Pediatricians with backgrounds treating pediatric patients with Rare Disease / Orphan Drug conditions
  • Global expertise in varied regulatory issues – critical for Rare Disease projects
  • ClinTrak, Medpace proprietary Study Management system, providing decision support for all aspects of a project including managing Late Phase Patient registries
  • Active member of the World Muscle Society, a non-profit organization dedicated to advancing knowledge in the neuromuscular field for over 20 years by providing a scientific forum to advance and disseminate knowledge for the benefit of patients