Rare Diseases

Where we meet every challenge with confidence.

Where we meet every challenge with confidence

With more than 20 years’ experience, we know better than most that rare diseases trials are different from any other. What may present as routine steps can have hidden challenges.

So everything we do is about helping you to not only overcome them, but to do that efficiently and in a bespoke way that maximize outcomes.

Aixial people. Your partners.

Our people around the world are highly qualified medical, scientific, regulatory and operational staff with extensive global drug development experience. They are supported by a network of KOLs and contacts. Each one is committed to and cares about making the most difference to the design and effective operational delivery of your rare disease trial in every way we can with:

Expert consultancy on business, regulatory issues, medical and scientific objectives. We can also offer a detailed review of a trial’s operational aspects.

In-depth and up-to-date knowledge of the global regulatory environment and legislation. That includes clinical trial supplies issues and the most appropriate solutions.

A global network of investigators who have a track record of high enrolment enhanced by internal and external database searches.

Ask us about:

  • Regulatory pathway selection
  • Patient enrolment and logistics
  • Country and site selection
  • Trial design
  • Medicinal product logistics
  • Data collection and delivery

Our exceptional experience of designing and delivering studies for many rare diseases includes these indications:


Alpha-1 Antitrypsin Deficiency (AATD)

Alpha – Mannosidosis


Autoimmune indications


Cluster Headache

Cushing’s Disease

Cystic Fibrosis

Eisenmenger Sydrome

Fabry Disease

Fragile X

Hereditary Angioedema

Hurler’s Disease

Inclusion Body Miositis

Inherited Metabolic Disorders

Lipoprotein Lipase Deficiency

Malignant rare diseases

Multiple Sclerosis

Myasthenia Gravis


Netherton Syndrome

Nevoid Basal Cell Carcinoma Syndrome


Noonan Syndrome

Paroxysmal Nocturnal Hemaglobinuria

PASLI Disease

Pediatric Hypertension

Pemphigus Vulgaris


Pigmented Villonodular Synovitis

Primary Biliary Cholangitis

Primary Biliary Cirrhosis

Primary Hyperaldosteronism

Pulmonary Fibrosis

Raynaud’s Disease

Rheumatoid Arthritis


Sickle Cell Disease

Sjogren’s Syndrome

Sjogren’s Syndrome

Spinal Muscular Atrophy (SMA)

Systemic Sclerosis


Type 1 Spinal Muscular Atrophy


Visceral Leishmaniasis

Wilson’s Disease

X-Linked Severe Combined Immunodeficency

How can we support your next project?