Give access to life saving medicine for terminally ill babies.

(3 Posts)
charlotterussell Sat 02-Apr-16 22:06:04

My daughter has SMA, diagnosed at 8 months. For the majority of children, this means that they are unlikely to live past their 2nd birthday. We, her parents, had never heard of it prior to our daughters diagnosis and yet 1 in 40 of us (mostly unknowingly) carry the faulty gene for it. 2 carriers have a 25% chance of conceiving a baby with SMA in each pregnancy. It is a devastating condition that manifests itself causing increasing weakening of the muscles until the sufferer no longer has the muscle strength to continue breathing. Our baby will never sit unsupported, crawl or walk. We've had to watch as she's lost her ability to move her legs and arms. Her mind is unaffected. But there is a drug that could halt and reverse these symptoms and all we are asking for is just a chance to access it for our daughter and thousands of other terminally ill children in her condition. As a mum and desperate and I'm begging for your help. I don't want to say goodbye to my baby girl! https://petition.parliament.uk/petitions/121253 . If you could spare a minute to read this petition I'd be so grateful and please see Helena and her fight at www.helpforhelena.CO.UK

Many thanks!

minatiae Sat 02-Apr-16 22:24:30

This drug has not completed clinical trials. It is in a Phase 3 trial, it is not being marketed. The laws regarding clinical trials and unapproved drugs cannot just be changed. This is the case for all clinical trials and all new drugs, the process is there for a reason. A government petition is unlikely to do anything.

What you really need to do, realistically, is to petition the company responsible for producing the drug to apply for the Early Access to Medications Scheme under the Medicines & Healthcare products Regulatory Agency. They will assess it for Promising Innovative Medicine status based on the Phase1/2 trial data, and it looks like this particular drug and condition is eligible for such status as per the website on this scheme.

https://www.gov.uk/guidance/apply-for-the-early-access-to-medicines-scheme-eams

Twowrongsdontmakearight Sat 02-Apr-16 22:35:15

I've signed for you and I truly hope that you manage to get treatment for your gorgeous daughter. flowers

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