Rare Diseases

A Wealth of Experience

We have collaborated with our clients on projects to research rare diseases, rare disease treatment, and complications and comorbidities of rare diseases.

Our experience includes:

  • Acromegaly
  • Amyotrophic lateral sclerosis
  • Angelman syndrome
  • Autoimmune pulmonary alveolar proteinosis
  • Autosomal dominant polycystic kidney disease
  • Central precocious puberty disease
  • Cushing's disease
  • Cystic fibrosis
  • Duchenne muscular dystrophy
  • Erythropoietic protoporphyria
  • Eosinophilic esophagitis
  • Fabry disease
  • Fragile X syndrome
  • Hereditary angioedema
  • Hunter syndrome
  • Hypoparathyroidism
  • Meniere’s disease
  • Myelofibrosis
  • Narcolepsy
  • Neurofibromatosis
  • Niemann-Pick disease
  • Paroxysmal nocturnal hemoglobinuria
  • Prader-Willi syndrome
  • Primary immunodeficiency disorder
  • Short bowel syndrome
  • Sickle cell disease
  • Spinal muscular atrophy
  • Sporadic inclusion body myositis
  • Wilson disease

Types of Projects

We have implemented studies to help our clients develop strategies in the rare disease market and to develop and gain market access for products to treat rare diseases and complications arising from treatment of rare illnesses.

Recent projects have included: