Neurology

Addressing this major unmet medical need in neurology

In 2023, projections predicted that the population suffering from neurological disorders would double by 2050². More recent data indicates that more than 3 billion people worldwide, which is to say over 30% of the global population, are affected by a neurological condition, making these conditions the leading cause of disease and disability³. Among them, neurodevelopmental and pediatric conditions alone account for almost 20% of the total neurological burden¹.

Despite the growing medical need, only a limited number of treatments are available to alter the progression of these disorders or alleviate symptoms significantly. We are committed to addressing this major unmet medical need in neurology with a particular focus on:

• Refractory epilepsies – These disorders primarily affect the central nervous system and are characterized by severe, recurrent seizures that resist standard treatments, leading to neurodevelopmental disorders or severe cognitive impairment. Although epilepsy is a common, serious neurological condition, 30-40% of patients experience uncontrolled seizures that resist appropriate antiseizure medications⁴.
• Rare movement disorders – These disorders primarily affect the central nervous system, impairing control and coordination of movement and have a severe impact on patient quality of life. They often emerge in childhood with symptoms of altered voluntary and involuntary movements. Examples include Friedreich ataxia and spinocerebellar ataxia^5,6.
• Neuromuscular disorders – These disorders primarily affect muscles and the nerves controlling them, resulting in muscle weakness, atrophy, and sensory disturbances like tingling⁷. Symptoms may begin in infancy or adulthood, vary in severity; some forms are life-threatening. Examples include amyotrophic lateral sclerosis (ALS) and generalized myasthenia gravis⁷.

What do all these disorders have in common? Patients with these neurological disorders exhibit symptoms that can begin in infancy and continue into adulthood. Although the symptoms vary, they are typically severe, causing disability, significantly reducing quality of life, and may even lead to death. With limited therapeutic options available, patients’ medical needs remain high. By focusing on these neurological disorders, we also target the shared biological pathways, creating multiple synergies to enhance our chances of success and speed up development of new medicines for patients.

By patients’ side

Depression

We have been working with the patient organization GAMIAN-Europe for many years. In 2022, we organized two patient workshops, which enabled us to collaboratively produce two brochures on the symptoms of depression and how to treat the condition.

• Anxiety symptoms in depression explains anxiety and offers advice to help people better cope with this symptom.
• Managing depression: A 3-step approach presents the three key steps in dealing with depression.

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Insight: Depression, the leading cause of disability worldwide

Often misdiagnosed, depression is a disorder that can have many causes, and there is a lot we still do not understand.