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A commitment to addressing major medical needs in rare diseases

In the U.S. alone, more than 25 million are impacted by rare neurological diseases. Globally, there are around 600 rare neurological disorders that have been identified to-date. Despite their “rare” status individually, their collective burden is massive. At Servier, we focus on creating new medicines that aim to slow down or halt the progression of rare neurological disorders and significantly improve patient quality of life.

Why entering neurology?

Building on our established leadership in oncology, we are expanding into rare neurological diseases with a clear ambition: to bring the same scientific rigor, focus, and partnership-driven approach to a new set of complex conditions.

Our experience in building a differentiated pipeline – combining cutting-edge science with strategic collaborations – positions us to accelerate innovation in neurology.

What are our focus areas?

We are advancing a focused pipeline in rare neurological diseases, targeting conditions where the unmet need is highest and where our scientific expertise can make a meaningful difference. 

To advance this pipeline, we leverage a range of therapeutic modalities – including small molecules, monoclonal antibodies and antisense oligonucleotides – selected based on the underlying biology of each disease. 

Our research is centered on a select number of shared biological mechanisms across diseases, enabling us to develop targeted strategies to address their progression, supported by patient-derived data and robust biological evidence. 

We are currently prioritizing three areas:

  • Rare refractory epilepsies refer to a group of uncommon epilepsy syndromes that are resistant to standard anti-seizure medications and often have a genetic origin. These conditions typically begin in infancy or childhood, are associated with severe seizures, and can lead to neurodevelopmental regression, cognitive impairment, and significant morbidity. Some forms are progressive and life threatening.
  • Rare movement disorders are an expansive and diverse group of uncommon neurological conditions, those with a genetic component often begin in childhood or early adulthood. They are marked by abnormal involuntary movements such as tremors, dystonia, chorea, ataxia or paroxysmal dyskinesias, and are frequently progressive and disabling with limited treatment options. Examples: Friedreich’s ataxia and spinocerebellar ataxia.
  • Neuromuscular disorders can be genetic, immune-mediated, or due to other causes that impair any component of the neuromuscular system, including the motor neurons, peripheral nerves, neuromuscular junctions, or muscle fibers. They can present progressive muscle weakness, muscle wasting, and functional impairment. These can be chronic and debilitating, and for many neuromuscular disorders, no curative treatment is available. Examples: amyotrophic lateral sclerosis and autoimmune myasthenia gravis.

This work is enabled by our One Innovation Engine approach – a model designed to integrate our internal R&D capabilities with external partnerships, diverse expertise and advanced technologies. Together, these elements allow us to accelerate the discovery, development and delivery of new treatments.