'Amazing' gene-silencing drugs reach NHS - (BBC News via NewsPoints Desk)

  • Gene-silencing therapies have been approved by NHS for the treatment of amyloidosis, BBC News reported Tuesday.

  • Patisiran was the first such treatment approved for this indication, while inotersen was authorised a few weeks ago.

  • Previously published data revealed that patisiran can halt or reverse the course of the disease.

  • "This is making a disease that was previously untreatable, treatable," remarked Julian Gillmore of the Royal Free Hospital.

  • "[Amyloidosis] is probably the first of many potential diseases that will be treated by gene silencing," Gillmore predicted, continuing "this has very far-reaching potential consequences, it has a huge potential."

  • "Today's announcement has the potential to change the lives of families across the UK who are affected by this cruel disease, offering them an option that could reduce the burden of symptoms that can be crippling to many," stated Carlos Heras-Palou of the ATTR Amyloidosis Patients' Association.

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