Spinraza, (not so) NICE, and SMA Type 1

Guest blog by Natasha Kelly, many thanks and respect to her:

Imagine taking your baby to the doctors to be told that they have a critical genetic disorder that will more than likely kill them before they reach their second birthday (just let that sink in for a moment).  

Now imagine part of that is to watch them lose the functions they need in order to live, their ability to swallow, to eat, to move at all, to breath before finally a common cold kills them!  

Now imagine they are completely aware as this disorder does not affect their brain at all, imagine the questions they would ask if they could and the heartbreak knowing their was nothing you could do.  

Now imagine the doctor saying their is a drug that could help, it wont cure your baby but it would stop the deteriorating and in some cases regain strength back for your baby, to eat, sit up and maybe one day walk!! (Fantastic right??)

Now imagine that your baby cant have that drug as NICE have decided its not worth the money! 😡 your baby is not worth the cost!! (Let that sink in)

This is what the SMA community are currently fighting! Please contact your local MP, come to the protest on the 7th September in Manchester or London, kick up a fuss with us, let them know our babies are worth it!! Whatever the cost!!

Many thanks again to Natasha and Elsie-Rose, now from me:

Spinraza is a medication that can be used to treat Spinal Muscular Atrophy, and is injected directly into the central nervous system. In clinical trials it has been found to halt the progression of the condition, indeed, in 60% of recipients with SMA Type 1 it has significantly improved motor function.

Although accepted for 'restricted' use in Scotland (website), the National Institute for Health and Care Excellence (NICE) has refused to support the NHS using Spinraza as a treatment in England (website). The opinion of NICE is that, although evidence provided showed a "substantial clinical benefit" it's cost is too high and there are a "a lot of uncertainties around its long-term benefits."

The protests mentioned above and organised by Treat SMA (facebook page for more details, website) are both planned for 11.30 am Friday 7th of September 2018 at the Department of Health, 39 Victoria Street, London SW1 0EU, and at NICE, Manchester City Tower, Piccadilly, Manchester M1 4BT. If you cannot be at either, we would be very grateful if you would contact your MP please; here for details. If you live in the Hastings & Rye constituency, Amber Rudd MP (details), or email her at amber.rudd.mp @parliament.uk

The young girl in question here, and local to Hastings, is Elsie-Rose (in the photograph above), a local fisherman's grand-daughter, and you may have noticed, lately I've been writing plenty about SMA Type 1 and the Elsie-Rose & Friends Foundation. If not, and you'd like to read more about both please have a look at this recent blog, many thanks, and many thanks for your support!

Don't forget #treatsma #smaawareness and #spinrazachangeslives