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Approve treatment to save Matty's kidneys before it's too late

This petition had 386,224 supporters


My son Matty, is a normal 15 year-old boy. He is intelligent, popular, loves to be active and has lots of friends.

Unfortunately he has a very rare and life-threatening condition called Dense Deposit Disease (DDD) which is slowly eating away at his Kidneys. Conventional therapies such as steroids have no effect on the condition and there is no recommended treatment available on the NHS.

Sometime soon Matty's Kidneys will fail and he will be forced on to dialysis and then he will need a kidney transplant. That's where the problems start though because - apart from the obvious risks of major surgery and organ rejection - the disease is likely to return and attack the new kidney.

There is a light at the end of the tunnel.

There is one drug available which can stop Matty's deterioration and allow him to live a normal life. Eculizumab is licensed for other rare diseases in the UK but doctors aren't allowed to prescribe this for Matty even though all the evidence is that it will stop this disease in it's tracks.

The problem is that Matty is caught in a Catch-22.

Doctors can't prescribe because there is no NHS policy for using Eculizumab for DDD. There is no policy because there are no clinical trials and there will never be any clinical trials because the condition is too rare. Now the NHS has a process for that - it's called an Individual Funding Request. Unfortunately the IFR process demands that patients are clinically exceptional and the assessment of that definition is made by a self-elected screening team without the right for clinicians to appeal and not subject to any external scrutiny or accountability. So because there are a handful of DDD patients they are all effectively banned from using the IFR route.

So the disease is too rare for a policy but not rare enough for individual treatment.

In case you are wondering whether the drug works consider this:

1) ALL - 100% - of the published case studies of patients with DDD with certain clinical markers (which Matty has) have responded to Eculizumab

2) Patients in Scotland (and many other patients in Europe) are being treated for DDD with Eculizumab.

3) Insurance companies in the US are paying for Eculizumab for DDD

Please sign this petition today and tell Jeremy Hunt MP and the NHS to correct their policy NOW and let Matty have this life-saving drug before it's too late.

You can follow Matty on Twitter: @Eculiz4matt 

and on Facebook: https://www.facebook.com/matty.jones.716970



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