26 Nov 2014

NZ scientist discovers Rett drug

8:43 pm on 26 November 2014

An Auckland University scientist has discovered a drug to treat a crippling brain disease that affects thousands of women worldwide.

Margaret Brimble and her team made the break-through that if approved will be the first New Zealand discovery registered by the United States Food and Drug Administration (FDA).

Prof Brimble said the FDA might fast-track its registration so it could be sold in one or two years, as no drug is currently available for sufferers of Rett syndrome.

Symptoms of the syndrome are similar to those of autism and cerebal palsy, and it affects about one in 12,000 girls. It also occasionally occurs in boys.

Prof Brimble said trials had already proved it was safe and worked, giving hope to sufferers who generally only lived only to about 35 years of age.

"It's a drug for a disease for which there is nothing for these patients," she said.

"So anybody who had a daughter or a female member of their family who suffered Rett syndrome would spend their whole life just willing people to come up with a drug."