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Our Approach to Drug Development

Our scientific curiosity, pioneering spirit and fundamental understanding of the conditions we seek to treat make it possible to take on the biggest challenges in rare disease, resulting in first-in-class or best-in-class therapies.

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Our Pipeline

We take on the biggest challenges in rare disease.

Each drug candidate we pursue is guided by a fundamental understanding of the genetics and underlying biology of the condition we hope to address.

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Clinical Trials

More than 20 years of proven clinical success.

Our thoughtful approach to clinical trial design enables us to move fast and efficiently while protecting the safety of our patients.

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Funding & Support

We support programmes and activities that foster excellence in patient care and provide valuable scientific, medical, and educational information.

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