Rare Diseases

We deal with the challenges, so you can focus on the opportunities

Not only due to a limited number of patients and qualified study sites, orphan disorders require unique approaches in study conduct. Knowing the exceptional challenges, we handle these projects with profound understanding of Orphan Drug Development.

Our core competencies:

  • Thorough protocol design taking into consideration a potential lack of literature
  • Searching and choosing motivated and experienced sites
  • Efficient patient recruitment
  • Acting as sponsor for EU Orphan Drug Designations

 

Therapeutic Approaches include:

  • Cystic fibrosis
  • Systemic lupus erythematosus (SLE)
  • Limbal stem cell deficiency (LSCD)
  • Glioblastoma
  • Neuroblastoma
  • Hepatocellular carcinoma
  • Fabry disease
  • Primary progressive aphasia
  • Haemophilia B
  • Head and neck carcinoma
  • Huntington’s disease
  • Advanced renal cell carcinoma
  • Malignant ascites
  • Primary sclerosing cholangitis
  • Acute-on-chronic liver failure (ACLF)
  • Multiple myeloma
  • Eosinophilic esophagitis
  • Ehlers-Danlos syndrome
  • Indolent systemic mastocytosis
  • Granulomatosis with polyangiitis (Wegener`s)
  • Acute myeloid leukemia
  • Friedreich’s ataxia
  • Schnitzler’s syndrome

Pediatric Studies in Orphan Diseases

  • Neuroblastoma
  • Limbal stem cell deficiency (LSCD)
  • Systemic lupus erythematosus (SLE)
  • Childhood cerebral adrenoleukodystrophy (CCALD)

If you’re interested in our broad experience in overcoming the hurdles of rare disease trials,  get in touch with us.