Tech Transfer eNews Blog

U of Auckland researchers await FDA approval of novel drug for Rett syndrome

By Jesse Schwartz
Published: March 15th, 2023

Researchers at the University of Auckland have developed a novel treatment for Rett syndrome, a severe neurological disorder affecting hundreds of thousands of girls and women around the world.

Rett Syndrome almost exclusively affects females, stalling or regressing their motor control and communication skills at six to 18 months of age. Margaret Brimble, researcher at the University of Auckland and co-developer of the drug, describes it as a “debilitating disease” and one still without an approved treatment.

Brimble and her team recently submitted the drug, known as Trofinetide, to the FDA. A decision is expected within days, and if approved it would become the first drug developed in New Zealand to be cleared by the U.S. regulator.

“To produce life-changing drugs for females, that’s a dream,” says Brimble. “I can now go to my grave knowing that I have used my science to try to help others.”

Source: NZ Herald

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