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Gene silencing medicine transforms crippling pain


An innovative type of medicine - called gene silencing - is set to be used on the NHS for people who live in crippling pain.

The drug treats acute intermittent porphyria, which runs in families and can leave people unable to work or have a normal life. Clinical trials have shown severe symptoms were cut by 74% with the drug.

While porphyria is rare, experts say the field of gene silencing has the potential to revolutionise medicine.

Sisters Liz Gill and Sue Burrell have both had their lives turned around by gene silencing. Before treatment, Liz remembers the trauma of living in "total pain" and, at its worst, she spent two years paralysed in hospital. Younger sister Sue says she "lost it all overnight" when she was suddenly in and out of hospital.

Both became used to taking potent opioid painkillers on a daily basis. But even morphine could not block the pain during a severe attack that needed hospital treatment.

Gene silencing gets to the root-cause of the sisters' disease rather than just managing their symptoms. Their porphyria leads to a build-up of toxic proteins in the body, that cause the physical pain. Gene silencing "mutes" a set of genetic instructions to block that protein production.

Both had been taking the therapy as part of a clinical trial and are still getting monthly injections.

The National Institute for Health and Clinical Excellence (NICE), which approves drugs for use in England, said the therapy "would improve people's quality of life" and was "value for money".

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Source: BBC News, 21 October 2021

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