Why It’s Important /

Why Do Research In Children?

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

When children are given a medicine, more than 70% of the time, 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.

This approach may sound like guesswork, but without research in children, it’s all we have. We need to think about how a child’s brain and body are developing…as well as the way a child’s body handles medicines and other treatments 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..."

Sawyer, child in Fabry disease study