Dame Margaret Brimble is already well known as one of New Zealand’s most distinguished scientists. But of all her professional achievements so far, the latest tops them all, she says. “This is the biggest,” the organic chemist says of the announcement last month that a drug based on a molecule she discovered more than two decades ago has received US Food and Drug Administration (FDA) approval.
The drug, trofinetide, is a synthetic version of a molecule essential for cognitive and motor function. In some people, a gene mutation inhibits formation of the molecule, causing a condition called Rett syndrome, which has symptoms that vary in severity, but are similar to cerebral palsy and autism. It almost exclusively affects females,
