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
  • Industry supporter of Rare Disease UK, an organization providing a united voice for the rare disease community by capturing the experiences of patients and families and working to raise the profile of rare diseases across the UK, seeking to bring about lasting change offering better health and quality of life for individuals and families affected by rare diseases