Focus Areas

Finding innovative solutions for unmet medical needs is at the heart of what we do. Our ideas and research help save lives.

This information is intended for the general public. Please consult your doctor if you need more information.

Spinal Muscular AtrophySolutions

SMA is an autosomal recessive genetic disorder caused by reduced levels of SMN protein throughout the body, resulting from mutations in the survival motor neuron-1 (SMN1) gene.

Close-up of numerous transparent lab tubes in colorful holders, including purple and red. The arrangement suggests a scientific setting.

CancerSolutions

Cancer remains the second leading cause of death in the industrialised world and its incidence is growing.

Multiple SclerosisSolutions

We’re committed to advancing the clinical understanding of underlying disease activity and progression for the millions of people living with multiple sclerosis (MS) worldwide.

Rheumatoid arthritisSolutions

Rheumatoid arthritis is a disease affecting more than 21 million people worldwide.

HaemophiliaSolutions

Haemophilia is a serious, inherited bleeding disorder in which a person’s blood does not clot properly. in severe cases leading to uncontrolled bleeding, either spontaneously or after minor trauma.

‘Retinal diseases’ are a broad range of eye diseases that affect any part of the retina, a thin layer of tissue at the back of the eye. The retina is involved in sending information to the brain, enabling sight