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:
Acromegaly
Alpha-1 Antitrypsin Deficiency (AATD)
Alpha – Mannosidosis
APDS
Autoimmune indications
Beta-thalassemia
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
Myositis
Netherton Syndrome
Nevoid Basal Cell Carcinoma Syndrome
NOMID/CINCA Syndrome
Noonan Syndrome
Paroxysmal Nocturnal Hemaglobinuria
PASLI Disease
Pediatric Hypertension
Pemphigus Vulgaris
Phosphomannomuttase
Pigmented Villonodular Synovitis
Primary Biliary Cholangitis
Primary Biliary Cirrhosis
Primary Hyperaldosteronism
Pulmonary Fibrosis
Raynaud’s Disease
Rheumatoid Arthritis
Sarcoidosis
Sickle Cell Disease
Sjogren’s Syndrome
Sjogren’s Syndrome
Spinal Muscular Atrophy (SMA)
Systemic Sclerosis
Tuberculosis
Type 1 Spinal Muscular Atrophy
Uveitis
Visceral Leishmaniasis
Wilson’s Disease
X-Linked Severe Combined Immunodeficency
How can we support your next project?
