Promising gene therapy delivers treatment directly to brain

Medical Research Cropped

The Associated Press

Scientists say delivering gene therapy directly to the brain holds great promise.

The first brain-delivered gene therapy on the market was recently approved in Europe and the U.K. for a rare genetic disorder called AADC deficiency.

It causes developmental delays and movement disorders in kids.

New Jersey drugmaker PTC Therapeutics plans to seek approval for the treatment in the U.S. this year.

Meanwhile, about 30 studies in the U.S. are testing gene therapy directly to the brain for other disorders, including Alzheimer’s and Parkinson’s.

Challenges remain, especially with diseases caused by more than a single gene. But scientists say the evidence supporting this approach is mounting.

Read the full story on AP news right here.

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1/24/2023 3:49:41 PM (GMT -6:00)

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