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Characterizing the patient experience: The role of patient‑reported outcomes

Biological endpoints are essential, but on their own, they cannot tell the full story. To truly understand the value of a treatment and care surrounding it, we must also seek to understand how patients and carers experience it. This is where patient-reported outcomes (PROs) and observer‑reported outcomes (reflecting the perspectives of non-clinical caregivers) are becoming more prominent in clinical research.

A bridge to the field: how real-world data informs rare disease care

As a Field Medical Advisor in Rare Disease, Antonios Letis works at the intersection of data, physicians, and patients. His focus includes cholestatic conditions such as PBC — areas where innovation has shifted what is possible.

Rethinking discovery: how powerful screening tools are redefining the search for new medicines

In medicine discovery, identifying the right biological target has always been a critical and complex step. Today, the rise of powerful screening tools is fundamentally reshaping this process, creating a step-change in how we move from scientific hypotheses to transformative medicines. Advances in high-throughput screening, genomics and AI-driven analytics are enabling scientists to interrogate disease biology at unprecedented scale, bringing forward opportunities that were previously beyond rea

Adult PFIC: Why Some People Spend Years Searching for Answers

PFIC is a group of rare inherited liver conditions that affects the body's ability to transport and circulate bile, which is essential for digesting and absorbing fats, as well as removing waste products.1–5 When bile builds up in the liver , it can damage the liver and cause symptoms such as itching.1

When science advances, understanding must keep pace

We are living in an era of extraordinary scientific progress. We can target diseases in ways that were unimaginable a generation ago, develop increasingly sophisticated medicines and explore entirely new approaches to treatment.

Building more sustainable laboratories with My Green Lab

Science is at the heart of everything we do. It also comes with an environmental footprint, from the energy and water used in laboratories to the chemicals, equipment and materials required to advance our work.