‘Major breakthrough’: first effective drug for Tay-Sachs successfully tested

Rare inherited disorder is more prevalent among children of Ashkenazi origin than the wider population

A PIONEERING NEW treatment for Tay-Sachs disease has shown positive results in a late-stage clinical trial, with charities hailing the "major breakthrough".

IntraBio, a US pharmaceutical company based in Oxford, announced the results last week of what it said was the “first successful clinical trial” for the treatment of Tay-Sachs disease.

The rare inherited disorder is more prevalent among children of Ashkenazi origin and affects the nervous system.

Tay-Sachs is usually fatal and its symptoms include seizures, loss of vision and paralysis and gets progressively worse over time.

IntraBio said it would continue to work with regulators and discuss “the exciting results" of its clinical trial.

“We believe the positive results of the trial speak for themselves, and are thrilled to continue to show the efficacy and safety of [the drug],” a spokesperson for the firm said.

The full results are set to be published in a peer-reviewed publication next month.

Principal investigator Dr Susanne Schneider, from Ludwig Maximilian University of Munich, said the “results were hugely important” for those affected by Tay Sachs and Sandhoff diseases.

“It is the first drug to offer hope to the patients and families affected by these devastating diseases,” said Cure Action for Tay-Sachs Foundation CEO chief Dan Lewi and Rick Karl, who heads the Cure Tay-Sachs Foundation.

FULL STORY ‘Major breakthrough’ as first effective drug for Tay-Sachs is successfully tested (Jewish Chronicle)