Accelerating Clinical Development in Rare Diseases – How can we be more efficient?

Kellogg College is pleased to host the Inaugural Lecture of Kellogg Fellow and Professor of Paediatric Neuromuscular Diseases, Professor Laurent Servais. The lecture will be presided by Kellogg President and Pro Vice-Chancellor, Prof Jonathan Michie.

When it comes to translating exciting pre-clinical discoveries in the field of rare and devastating diseases in the brains and muscles of children into a disease-modifying therapy in humans, the question “How can we be more efficient?” is a key concern for any physician involved in the process.

Too often, early findings in humans cannot be confirmed in large trials, leading to major disappointment in the field and a waste of time, money, human resources, and most importantly, of hope.

In this lecture we will explore together how we can improve efficiency now.

Innovative technologies are enabling us to accelerate clinical trial development, by demonstrating the presence or absence of an objective improvement on motor function in a limited period of time on a limited number of patients. New clinical trial design and statistics are allowing us to compare patients with their own evolution, rather than with another patient. And new born screening is empowering us to treat patients before irreversible damage, and to maximize the therapeutic effect.

Combining these different approaches has the potential to accelerate successful clinical developments, to avoid progressing drugs that have no objective efficacy into large trials, and even more significantly in the case of new born screening, to offer babies affected with severe but treatable conditions the best chance of having a life free of disability.

What is an Inaugural Lecture?

When an academic is promoted or appointed to the post of professor, and occasionally reader, in higher education, they are usually expected to deliver an initial lecture which is open to all, and which allows them to introduce and expound upon their existing body of work or their current research in their specialist field. This event is referred to as an inaugural lecture.

This in-person event will be taking place in the Kellogg Hub, as well as being streamed live. It will also be recorded and there will be a photographer present.

About the speaker:

Laurent Servais, MD, PhD is professor of paediatrics neuromuscular diseases at the University of Oxford and invited Professor at the University of Liege, Belgium. He graduated from the University of Louvain (Medicine) and Brussels (Paediatrics), and trained as a child neurologist in Robert Debré Hospital (Paris) and as a myologist in the Institute of Myology, in La Pitié Salpétrière (Paris). His main research interests cover innovative outcome measures and clinical trials design and newborn screening. He has been involved as PI in several clinical trials in spinal muscular atrophy, X-Myotubular Myopathy and Duchenne Muscular Dystrophy, and in leading the pioneering newborn screening program for spinal muscular atrophy newborn screening in Belgium.

Refreshments will be served from 5 pm in the Kellogg Hub, followed by the lecture at 5.30 pm.

If you are booking to attend online, you will receive a link with your confirmation email. If you book to attend in-person, but cannot make it, or if you do not receive the link to attend online, please email