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

Elsie-Rose


Recently I've been writing a number of reports about events that have centred around Mark Little, his kayak adventure (to be continued in approximately 10 days time!), and raising money for the Elsie-Rose & Friends Foundation (Crowdfunding).

Young local character Elsie-Rose, and this 4 year-old local lass certainly is a great character, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1 in September 2014, a condition that causes severe muscle weakness and results in problems with movement, eating, breathing and swallowing. Without intervention, sadly, most children with SMA Type 1 die within the first 2 years of life. However, the cheerful and resilient Elsie-Rose strives to carry on enjoying life just like any other 4 year-old child, despite all the complications she just keeps on smiling!

Elsie-Rose angling recently

Because many of the expenses encountered supporting children with SMA Type 1 are not provided for by the NHS, Social Services or the Department of Health, the Elsie-Rose & Friends Foundation was set up to support families with disabled, sick and terminally ill children. Indeed, my respect and warm regards go out to Elsie-Rose's mother, family and friends, and Elsie-Rose herself, and how they cope with the myriad of complications involved in their lives.

If you would like to know more about Elsie-Rose and the Elsie-Rose & Friends Foundation, and campaigns they are involved in, please go to their facebook page or twitter page. If you would like more information about Spinal Muscular Atrophy Type 1 please go to the Spinal Muscular Atrophy Support UK website.

Planet Hastings Crazy Golf & British Championships 2018 AND

OK, a library photograph! 

If you want to play on the Pirate Golf, one of the 3 courses at Hastings Adventure Golf, this weekend (website), you may well have a problem, no, not snowfall! That's a library image of mine (pretty picture), however, there may well be some light rainfall this afternoon and tomorrow (Sunday) morning... Oh yes, back to the point, this weekend Hastings hosts the British Championships 2018 (website), so expect congestion or, more likely, the Pirate Golf course will be closed to non-competitors!

Talking about Planet Hastings Crazy Golf Club reminds me of the recent generous donation they gave to Mark Little, contributing towards his 4-person team and their crossing the English Channel by kayak challenge in 3 months time (August). They will be travelling from Dungeness to Boulogne (Mark et al, not the 2 Davids and Maz) in aid of raising funds for the Elsie-Rose & Friends Foundation.

Local lass, young Elsie-Rose, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1 in September 2014, a condition that causes severe muscle weakness and results in problems with moving, swallowing, consequently eating, and breathing. Without appropriate intervention, most children with SMA Type 1 die within the first 2 years of their lives sadly. Thankfully, the resilient Elsie-Rose is determined to carry on and enjoy her family life just like other young children of her age!

Elsie-Rose

Because many of the expenses involved in supporting children with SMA Type 1 are not provided by the Department of Health, NHS, or local services, the Elsie-Rose & Friends Foundation was set up to 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 that they would raise funds for Elsie-Rose and the Elsie-Rose & Friends Foundation by kayaking across the English Channel.

If you would like to know more about the Elsie-Rose & Friends Foundation you can go to their facebook page or twitter page. If you would like to read more information about SMA Type 1, go to the Spinal Muscular Atrophy Support UK website. 

If you would like to make a contribution towards supporting this courageous wee lass and the Elsie-Rose & Friends Foundation please make a donation via Crowdfunding. Or you could visit The Dolphin Inn, 11-12 Rock-a-Nore Road, Hastings TN34 3DW, and make a pledge to support the team's kayak challenge, and/or add to the collection box on the bar, indeed, all contributions would be very much appreciated, many thanks!

Mark training for Channel Challenge, Elsie-Rose & SMA Type 1

As I've mentioned a number of times now, Mark Little and 3 other Team members are going to cross the English Channel by kayak in August, leaving from Dungeness, and landing in Boulogne several hours later, no doubt with aching arms! Why? Well, to help raise funds for local lass Elsie-Rose, the Elsie-Rose & Friends Foundation, and to ensure greater awareness of SMA Type 1.

Elsie-Rose was diagnosed with SMA (Spinal Muscular Atrophy) Type 1 in September 2014, a condition that causes severe muscle weakness, and creates problems with movement, breathing and swallowing. Sadly, without intervention, most children with SMA Type 1 die within the first two years of their shortened lives. However, with her courageous and strong character, Elsie-Rose strives to enjoy life just like any other 4 year old!

Mark practicing out there!

The Elsie-Rose & Friends Foundation was also set up to provide help and financial support to families with disabled, sick, and terminally ill children. After meeting Elsie-Rose and members of her family, Mark and his team decided to take up the challenge and kayak across the English Channel with the aim of raising money for Elsie-Rose, and for the Foundation.

Consequently, they are seeking donations via Crowdfunding to go towards the cost of any special equipment Elsie-Rose may need, and to provide the Elsie-Rose & Friends Foundation with funding and to support other families in need. There is also a collection box and sponsorship forms at the Dolphin Inn, 11-12 Rock-a-Nore Road, Hastings TN34 3DW.

A wee bit closer

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

To help support the courageous young Elsie-Rose and others, a donation via Crowdfunding would be very gratefully received, or visit the Dolphin Inn and make a pledge to support the Kayak Team, or add to the collection box, many thanks!

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, 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!

Raising Money for Elsie-Rose and SMA Type 1

The landlord of the Dolphin Inn, Mark Little, and 3 other hardy volunteers, will be taking on the challenge of kayaking across the English Channel from Dungeness to Boulogne in August to support a local lass, 4 year-old Elsie-Rose and SMA Type 1. 

Elsie-Rose was diagnosed with Type I SMA (Spinal Muscular Atrophy) in September 2014, a condition that causes severe muscle weakness, and which can result in problems moving, eating, breathing and swallowing. Unfortunately, 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 just like any other 4 year old! 

The Elsie-Rose & Friends Foundation was set up to provide help and financial support for families with disabled, sick, and terminally ill children and, after meeting Elsie-Rose and her family, Mark and friends decided to kayak across the English Channel with the aim of raising money for Elsie-Rose and the Foundation.

Consequently, they are seeking donations to their cause via Crowdfunding towards equipment Elsie-Rose may need in the future, and to give the Foundation funds it needs to support other families in need. Indeed, £350 has already been donated, including £250 from a kind anonymous donor! There will also be pots for donations and sponsor 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 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 help support this brave young lass and others, a donation either via Crowdfunding or by visiting the Dolphin Inn and making a pledge to support the team's kayaking challenge, or to add to the collection pots, would be gratefully received, many thanks.