More than half of epilepsies start before the age of 20 years and nearly 25% of them are refractory.  A tiny proportion of them are even labelled a "rare illness", i.e. one affecting less than 200,000 people in the United States or less than 5 out of 10,000 people in the European Union.  For these patients, two problems in their management arise:  In children, seizures affect an immature brain and can impact its development. It is therefore essential to treat them so that brain development continues as normally as possible. The rarity of these syndromes does not encourage the pharmaceutical industry to invest in research.   Therefore, there are few drugs available.  However, a few target syndromes with a particularly unfavourable diagnosis. This work outlines 6 molecules in detail that are specifically used in epileptic encephalopathies. Every aspect of these molecules is discussed, including drug development, indication, efficacy, cost, etc.  Drafted by international experts in the field of epileptology, it provides all the necessary information on orphan drugs and their clinical use.

Orphan Drugs in Epilepsy

More than half of epilepsies start before the age of 20 years and nearly 25% of them are refractory.

A tiny proportion of them are even labelled a "rare illness", i.e. one affecting less than 200, 000 people in the United States or less than 5 out of 10, 000 people in the European Union.

For these patients, two problems in their management arise:

- 

In children, seizures affect an immature brain and can impact its development. It is therefore essential to treat them so that brain development continues as normally as possible.

- 

The rarity of these syndromes does not encourage the pharmaceutical industry to invest in research.

Therefore, there are few drugs available.

However, a few target syndromes with a particularly unfavourable diagnosis. This work outlines 6 molecules in detail that are specifically used in epileptic encephalopathies. Every aspect of these molecules is discussed, including drug development, indication, efficacy, cost, etc.

Drafted by international experts in the field of epileptology, it provides all the necessary information on orphan drugs and their clinical use.

Epileptic encephalopathies have been discussed in different publications but mainly in relation to childhood. However, recent years have shown an increase of the life expectancy and social integration of patients with epileptic encephalopathies. Although the treatment goals remain the same regardless of age, the clinical symptoms, EEG-abnormalities, mental and behavioural aspects, side effect profiles of antiepileptic drugs change over time, requiring evolving and sometimes innovative therapies. Moreover, the social issues are important to consider for the maintenance of consistency in the delivery of care.

The present book covers the long-term evolution of epileptic encephalopathies in terms of clinical symptomatology, cognitive functions, treatment strategies and social care options. It will help clinicians, healthcare practitioners and social service professionals to understand better the natural history of epileptic encephalopathies, to identify specific management issues and to develop appropriate care strategies for this category of patients.

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