Rare Disease & Pediatrics

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Wide experience in rare and orphan diseases

Excelya Study Medical Managers steer comprehensive studies for our clients in many rare diseases, such as Lysosomal Storage Diseases with enzyme replacement and substrate reduction therapies for adults and children.

Clinical research in this field sets extra challenges involving site selection, identification and enrolling a limited number of patients. These include paediatric cases needing specific protocols, technical support and committed expert teams to deliver positive outcomes.

This is why Excelya also works with patient associations, boards and committees. Its aim is to deliver secure, reliable and quality clinical research studies allowing our clients to accelerate market launches in their strategic niches and thereby change patients’ lives.

  • Patient recruitment
  • Guaranteed secure pathology diagnosis
  • Biological follow-up
  • Biostatistics and biomarker expertise
  • Patient centricity