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Our group aims to: conduct and design best clinical trials in neuromuscular diseases; accelerate clinical trial development through innovative outcome measures and newborn screening; understand progress of the disease and apply personalised care; connect with families and act as a strong reference point; assess effectiveness of current treatment therapies; minimise undiagnosed cases and time to targeted intervention.

STRONG team photo, featuring Chris Montague-Johnson, Charlotte Lilien, Jenny Riga, Laurent Servais, Dom Tromans, Sithara Ramdas and Beatrice Yeung

Group Head Biography

Laurent Servais is Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre and Invited Professor of Child Neurology at Liège University.

After graduating from Louvain Medical School, Brussels, Belgium in 1999, he completed a PhD in Neuroscience (cerebellar electrophysiology in alert living mice) from Free University of Brussels, Belgium, followed by residencies in child neurology at the Free University of Brussels and Robert Debré Hospital, Paris. In 2008, he took a position in neuromuscular disease and clinical research at the Institute of Myology in Paris, where his interest and expertise in neuromuscular diseases flourished. He was subsequently appointed Head of Clinical Trials and Database Services. Most recently, he served as Head of the Institute of Myology’s I-Motion (Institute Of Muscle-Oriented Translational Innovation), and Head of the Neuromuscular Centre in Liège, Belgium. He has joined MD UK Oxford Neuromuscular Centre and the University of Oxford in September 2019.

Laurent has been involved as principal investigator in numerous clinical trials to test treatments for Duchenne muscular dystrophy and spinal muscular atrophy (SMA).

He is the leader of the newborn screening program for SMA in southern Belgium. His main research expertise covers the development of innovative outcome measures, including connected devices for real-life patients’ evaluation. He was instrumental in the qualification of the first digital outcome by a regulatory agency, the SV95C in Duchenne Muscular Dystrophy

 

Research Summary and Aims

1. Clinical trials in the neuromuscular field:  

Our aim is to conduct in the best condition possible phase 1-3 clinical trial in neuromuscular diseases. We consider our responsibility to adopt as soon as possible the current advances in biomedical research and technology in the design of accurate and effective clinical trials. The utmost goal for our team is to be able to assess the upcoming treatment therapies based on sensitive and disease-specific measures.

 

List of Trials

Trial Name

Sponsor

Status

Clinical Trial Database

SIDEROS

Santhera

Recruiting

https://clinicaltrials.gov/ct2/show/NCT02814019

ESSENCE

Sarepta

In set-up

https://clinicaltrials.gov/ct2/show/NCT02500381

MOMENTUM

Sarepta

In set-up

https://clinicaltrials.gov/ct2/show/NCT04004065

MILOS

Santhera

Feasibility to be confirmed.

TBC

CLIQ-SMA

University Hospital Basel

Feasibility to be confirmed.

TBC

 

2. Understanding of natural history on neuromuscular diseases: clinical trial readiness

After having raised international natural history study in Spinal Muscular Atrophy and in X-linked Myotubular Myopathy, our group is currently working on an international natural history in Nemaline myopathy (Insert the link to ENMC Workshop) and in Angelman Syndrome. We consider this the cornerstone of understanding the progression of the disease and creating a strong reference point for patients, families and healthcare centers.

 

3. Innovative outcome measures: effective assessment & personalised

Innovative outcome measures such as wearable devices have the potential to dramatically decrease the trial duration and the number of patients needed to conduct the trials. The aim of our group is to pursue the validation of innovative outcome measure as measured from wearable (link to EMA qualification of SV95C).

 

4. Newborn screening & new genetic assays: driving research into early identification, potential treatments, and early intervention for rare disorders.

In the light of all the recent advances in the field of genetics and genetic therapies, the need for early intervention is unprecedent. The aim of our multidisciplinary team is to develop accurate and sensitive newborn screening tests, which will ultimately be accepted onto the National Screening Programme

 

Our team

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