Rare/Orphan Diseases

Apex has had great success conducting trials for rare diseases including ataxias, Huntington’s disease, and sickle cell anemia.

Utilizing Sponsor relationships with academic centers, advocacy groups, and community organizations, we have met or exceeded enrollment goals on all rare disease studies to date and delivered quality data to help advance compounds through development.

Disabled man on wheelchair

Indication experience:

  • Friedreich Ataxia
  • Spinocerebellar Ataxia (SCA)
  • Sickle Cell Disease
  • Huntington’s Disease

Specialized Services and Capabilities

  • Phase I-IV
  • Complex biomarker studies (Imaging, Electrophysiology, CSF sampling, etc.)
  • Adaptive trial designs
  • Elderly Populations
  • Eye tracking, balance assessments


Therapeutic Areas

We have an outstanding track record of success in the conduct of Phase I-IV trials across the central nervous system and internal medicine disorders.