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

Elsie-Rose & Friends Foundation Fundraising Update.

Following Natasha Kelly's successful completion of the Hastings Half Marathon on Sunday and raising nearly £1,500 for the Elsie-Rose & Friends Foundation (blog), this is an update on how the fundraising is going for Mark Little and the 3 other volunteers in his team, who will be kayaking across the English Channel from Dungeness to Boulogne in August (blog). They will also be fundraising in support of the local 4 year-old with Type I SMA (Spinal Muscular Atrophy), Elsie-Rose, whose grandfather and other family members work in the local fishing industry, and the Elsie-Rose & Friends Foundation. 

Young Elsie-Rose was diagnosed with Type I SMA in September 2014, a condition that causes severe muscle weakness, and results in problems moving, eating, breathing and swallowing. Without intervention, most children with Type I SMA die within the first 2 years of life, however, the resilient Elsie-Rose strives to carry on and enjoy life just like all other 4 year olds, or more so! 

Because many of the expenses involved in supporting children with Type I SMA are not provided by the Department of Health or NHS, the Elsie-Rose & Friends Foundation was set up to help provide help and financial support to families with disabled, sick, and terminally ill children. After meeting Elsie-Rose and her family, Mark and his team decided to kayak across the English Channel with the aim of raising money for Elsie-Rose and the Foundation.  

Donations are being sought via Crowdfunding which will go towards necessary equipment for Elsie-Rose and her carers, and to contribute funds towards the Foundation so that it may support other families in need. So far £440 has been donated via Crowdfunding, many thanks! There is also a collection box for donations and sponsorship forms in the Dolphin Inn at 11-12 Rock-a-Nore Road, Hastings TN34 3DW. 

If you wish to know more about Elsie-Rose, the Elsie-Rose & Friends Foundation and their campaign, you can go to their facebook page or twitter page. If you would like more details about SMA Type 1, go to the Spinal Muscular Atrophy website. 

To make a contribution towards supporting this brave 4 year-old and other children with similar problems, please make a donation via Crowdfunding or go and visit the Dolphin Inn and make a pledge to support the team's kayaking challenge, or add to the collection box, indeed, all contributions are very gratefully received, many thanks!

Hastings Half Marathon 2018 Results

It was cold, and with a strong wind racing in from the East, presumably Putin's response to Boris's bluster, only made it feel colder, but luckily it didn't start snowing until the half marathon was over... Anyway, here they are starting, I didn't see whether that photographer to the left got knocked over in the rush, he deserved to, leaving it so late!

The early leaders race by me, some blurred in their flight, or it could just be I'm not a proper photographer with an expensive camera, but you do get the feeling of speed...

Here was my final (regarding the race) resting place, my hands got so cold that my clapping made no noise, I hadn't realised that was what happened before! Anyway, here, the car that led them around the course passes by me, and it's 56 minutes and 11 seconds after the first photograph above...

Swiftly behind the car came the man who would finish first, Adam Clarke of Hastings AC, who finished in 1 hour 8 minutes and 35 seconds, CONGRATULATIONS!

Second, and a wee bit blurred, sorry, was to be this man, Charlie Joslin-Allen of Tonbridge AC, finishing in 1 hour 8 minutes and 52 seconds, a close finish!

Third was another Hastings AC runner, Gary Foster, the first of these 2 men, who finished in 1 hour 10 minutes and 59 seconds, 2 seconds ahead of the man behind him!

The first woman to cross the finishing line was Maria Heslop of Tonbridge AC in 1 hour 21 minutes and 28 seconds, CONGRATULATIONS for winning the women's race!

The second woman to cross the finishing line was another Hastings AC runner, Stacey Clusker, in a time of 1 hour 23 minutes and 37 seconds.

And the third woman to cross the finishing line in 1 hour 25 minutes and 52 seconds was yet another Hastings AC runner, Rachael Grass, nice one!

For the results, go to the website, apologies if I've made any errors, though I sincerely hope I haven't, but it appears 2,469 courageous runners finished the half marathon in bitterly cold conditions, which I believe may be a record number, if I heard the speaker correctly at the beginning. Natasha Kelly, who ran in support of the Elsie-Rose & Friends Foundation, finished in a very creditable time of 2 hours 27 minutes and 41 seconds, well done to her, and many thanks for her support of the Foundation, for which she raised nearly £1,500!

Indeed, my warm congratulations to all those who took part, softy me had to nip into the Cinque Ports Arms opposite to warm up my hands!

Hastings Half Marathon 2018, Today's The Day!

Well, today's the day for the 34th Hastings Half Marathon (website), starting at 10.30 at Sea Road, by Grosvenor Gardens, St Leonards, and finishing there some time later, and the weather doesn't look to be this inviting...

Many runners will be raising money for various charitable causes, including Natasha Kelly (Elsie-Rose & Friends facebook page), who is running to raise money for the Elsie-Rose & Friends Foundation: see my recent blog about the kayaking event across the English Channel to raise money for Elsie-Rose and the Foundation, and which also gives information about Elsie-Rose and Type 1 SMA (Spinal Muscular Atrophy). I am supporting this event too, and here is a link to the Crowdfunding Page which has raised £420 so far towards the Foundation, many thanks to all who have contributed. All donations to either Natasha and her run, or Mark and his team and their channel crossing will be deeply appreciated, cheers!

For more details of the route and map and warm up advice go to the website.

My best wishes to Natasha, the Hastings Lifeboat runners raising money for many charities, and everyone else who bravely runs the Hastings Half Marathon today, and make sure you wrap up warm if you're a spectator!