Rare Diseases
Where we meet every challenge with confidence.
With more than 20 years’ experience,
Aixial understands rare disease trials are unique.
Our rare disease experienced teams’ will provide proactive risk identification and mitigation specific to your target indication.
Aixial team, your trusted partner
Our global team consists of highly qualified medical, scientific, regulatory, and operational professionals with extensive experience in global drug development. They are backed by a network of Key Opinion Leaders (KOLs) and industry contacts. Each team member is dedicated to making a meaningful impact on the design and effective operational delivery of your rare disease trial, ensuring success in every aspect, including:
- 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 logitics
- Trial design
- Country and site selection
- Medicinal product logistics
- Data collection and delivery
Helping you suceed in your project
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 support your next project?
Whether you’re looking for a protocol review or a proposal,
simply reach out to us by filling our request for proposal.
