ABOUT DRAVET SYNDROME
Dravet syndrome (sometimes called severe myoclonic epilepsy of infancy or SMEI) is a debilitating form of epilepsy that begins in infancy. The condition is very rare, affecting only 1 in every 20,000-40,000 births and can result in:
- Behavioral and developmental problems
- Movement and balance issues
- Language and speech problems
- Growth issues
- Sleeping difficulties
- Frequent infections
- Nervous system problems
Certain anti-seizure medications have helped some children and young adults reduce the number of seizures they have. But they don’t work for everyone. That’s why studies (like those in the FAiRE program) are so important.
ABOUT CLINICAL RESEARCH
Clinical studies (also known as ‘clinical trials’) help researchers test new medications and improve (or test new uses for) those that are already available. They do this by asking participants to follow strict dosing plans and then comparing the results.
In this way, researchers can answer questions such as:
- Does this investigational drug work?
- Does it work better than another medicine already available?
- Does it cause any side effects?
These dosing plans and questions are laid out in the study’s ‘protocol’. A large group of people (including doctors, researchers and other specialists) review this protocol before the study begins. It is their job to protect the safety of participants. But the exact risks of a study can be hard to predict. So participants must agree to a schedule of clinic visits and/or telephone appointments so that the study team can monitor their health and learn how their dosing plan is affecting them.