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16
Jul 2019
‘Gene-silencing’ medicine approved for use by NHS
A new ‘gene-silencing’ drug has been approved for use by the NHS in England.
Doctors say the drug will make the ‘previously untreatable, treatable’ and has significant potential in medicine.
The gene-silencing treatment will be used to reverse a potentially fatal disease by the name of amyloidosis, which causes damage to organs and nerves.
There are different forms of the disease, a ‘rogue gene’ which leads to a build-up of toxic proteins in the body. The protein damages nerves and organs and can be fatal within three to 15 years of first symptoms, currently around 150 people in the UK are affected.
Professor Julian Gillmore of the Royal Free Hospital in London, said “This is making a disease that was previously untreatable, treatable.
“I’ve been watching these patients get worse for 25 years, I know families that have been devastated by this disease. This has the potential to change that, it’s amazing, it’s exciting.”
Studies have also shown that the treatment is effective in other genetic and inherited diseases, such as porphyria.
A trial drug has also shown in early stage research, that the defect which causes the neurodegenerative disease Huntington’s has been rectified with gene-silencing.
There are also ideas that the treatment could tackle other neurodegenerative diseases such as Parkinson’s or Alzheimer’s, and also dangerously high levels of cholesterol that runs in families, although this research is still in the early stages.
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Posted by Karen Motley, Clinical Negligence Department, Chadwick Lawrence LLP (tonymay@chadlaw.co.uk ), medical negligence lawyers and clinical negligence solicitors in Huddersfield, Leeds, Wakefield and Halifax, West Yorkshire.
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