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Researchers and families talk about why research in children is important.

Why Do Research In Children?

Medicines, devices and treatments are often not tested in children.

At nearly half of medical visits, children are given a medicine and 70% of those medicines have only been tested in adults.

The simple truth is...children are not little adults.

But without research in children themselves, we have no choice but to treat them that way.

Doctors and nurses often give medicines to children even though they have not been studied and approved by the Food and Drug Administration (FDA) for use in children. This is known as "off-label" use. Most of the time, this works well but when the adult dose is adjusted to the weight of a child, there is a chance that the dose used could be ineffective or even harmful.

While it may sound like "guesswork," it's really been more of a 'hand-me-down' approach. Without research in children though, it's all we have. We need to think about how a child's brain and body are well as the way that medicines and other treatments are handled in a child's body over time.

"If we can find a way to reverse some of the effects of the disease, we can make it better and maybe parents won't have to worry about it..."
child in Fabry disease study

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