Rare Disease

Our global reach allows us to study ‘hard to reach’ patients in 80 countries. Each project bringing us a step closer to understanding – and beating – rare diseases.

Over the past 10 years, we’ve delivered:

  • More than 40 rare and ultra-rare global studies
  • Fast access to over 190 sites and 14,000 patients
  • An additional 27 pediatric global studies

We specialize in pediatric, rare and ultra-rare diseases.

We share your passion for orphan drugs. Over 50% of rare diseases occur in pediatric populations. Our expert teams can provide support and solutions for the extensive requirements involved.

  • 40+
    Rare and ultra-rare global studies
  • 14k
    Patients over 190 fast access sites
  • 27
    Additional pediatric global studies

Flexible approach
The study of rare diseases requires a unique approach. We’ve refined referral networks from country to country to ensure fast access to the right patients for your study. We’ll work with you every step of the way to drive essential data to key decision makers across the globe.

Study structures
We approach every project as a blank canvas. Our customized monitoring models and study structures take account of local cultures, regulations and practices.

Personal approach
Rare disease trials can involve challenging patient considerations. We offer concierge services to ease travel burdens, particularly on high enrolling sites.

Global insight
Accumulated research throughout our network is helping us to build a clearer picture of the prevalence of rare disease across the globe. This insight lets us target specific regions and implement more efficient – and indeed, cost-effective – strategies.

Below is an example of some of the recent  rare disease programs we have been working on:

  • Gaucher’s Disease
  • Giant Cell Arteritis
  • Hookworm Vaccine
  • LAL Deficiency
  • Leishmaniasis
  • Mesothelioma
  • Niemann-Pick type C Disease
  • Sjogren Larsson Syndrome
  • Wolman’s Disease (LAL Deficiency)

We have also been working in a number of rare Oncology Indications

  • Acute Lymphoblastic Leukaemia
  • B-Cell Lymphocytic Leukaemia
  • B-Cell Lymphoma
  • Chondrosarcoma
  • Chronic lymphocytic leukaemia (CLL)
  • Chronic Myelogenous Leukaemia
  • Cystic Fibrosis
  • Gaucher’s Disease Type 1
  • Haemophilia A
  • Haemophilia B
  • Malaria
  • Mantle Cell Lymphoma
  • Multiple Myeloma
  • Multiple System Atrophy
  • Non-Hodgkin’s Lymphoma
  • Relapsed Osteosarcoma or Ewing’s Sarcoma
  • Sarcoidosis

Get in touch to find out more about our clinical trials in rare diseases

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