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Drug Shows Promise for Rare Genetic Disorder

New therapy may combat amyloidosis, the potentially deadly disease caused by protein build-up in organs

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For example, he added, regulators will want to see a reduction in organ damage or an overall improvement in survival rates.

Vaishnaw said he expects to be able to show those kinds of results, given that previous studies have shown that amyloid deposits will begin flushing from a person's organs if the levels of amyloid in the bloodstream decrease dramatically.

"It's allowing the organs to clear the deposits that are already there," Vaishnaw said. "We're hoping that over time we'll allow clearing of the existing deposits, which has been seen in other amyloidosis disorders."

However, it will likely be years before the medication passes through drug trials and receives FDA approval, he added.

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