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Sam and Fiona Bailey Órla Ryan

Spinraza campaigners 'finally have hope' as HSE approves access to drug for rare disease

The drug has been approved for children with SMA Type I, II or III on “an exceptional and individualised basis”.

THE HSE HAS approved access to the drug Spinraza in the treatment of children with spinal muscular atrophy (SMA) after a lengthy campaign by families and patients.   

SMA is a rare, debilitating and life-threatening disease that affects nerves in the spinal cord, making muscles weaker and causing problems with movement, breathing and swallowing. 

Where it develops in babies and toddlers it can significantly reduce life expectancy. While it is not a cure, trials have shown that Spinraza, also known as nusinersen, can slow the effects of SMA in some cases.

Minister for Health Simon Harris announced this morning that the HSE would fund the drug for children only. 

“This is the first ever treatment for children with SMA. I know how hard the long journey to get here has been for families but very glad children in Ireland will now have access,” Harris tweeted. 

In a statement today, the HSE said it had approved access for children with SMA Type I, II or III on “an exceptional and individualised basis”.

“The recommendation is to approve access for children under 18 years with genetically confirmed SMA Type I, II or III, in accordance with the controlled access criteria recommended by the Rare Diseases Technology Review Committee.”

To date, more than 7,500 patients worldwide have been treated with nusinersen, from infants to adults.

The most common form of the disease is 5q SMA, which has four different types: 1, 2, 3 and 4– based on age of onset and the highest physical milestone achieved. Type 1 is the most severe form of SMA and accounts for between 50–70% of cases of childhood onset SMA. About 95% of children with Type 1 who are untreated die before the age of two. 

Fiona Bailey, whose son Sam (9) has SMA, said they are all “absolutely over the moon” at today’s news. 

“It’s an amazing day for us all, it’s a day we’ve been working towards for a long time.

“The happiest day we’ve had in a long time, we finally have some hope for Sam’s future. Students all over Sam’s school were crying this morning after hearing the news,” Fiona told TheJournal.ie. 

Sam is stronger than anyone I know. In his heart and in his soul he is a fighter. Now that Spinraza has is a reality for us the future looks brighter. 

Concern 

The HSE had previously said it would not reimburse for the drug at its current price, stating it would cost more than €20 million over a five-year period.

The drug was approved by the NHS back in May after an agreement with its makers, Biogaen, to fund treatment for a limited time to allow more data to be collected on its effectiveness. 

In today’s statement, the HSE said it will work closely with the Department of Health in relation to the funding implications of today’s decision; particularly in relation to funding for future years.

SMA Ireland had previously voiced its concern that the HSE would apply an age-based cut off to treatment to save costs.

“We genuinely hope that reason and common sense has held sway and that treatment will be available for all people with SMA,” the group tweeted earlier. 

With reporting from Órla Ryan 

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    Mute Clodagh Carroll
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    Oct 5th 2013, 11:15 AM

    Bye and thanks for the many laughs on here, Capt. Adebayo, you legend!!

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    Mute Lloyd Christmas
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    Oct 5th 2013, 12:54 PM

    He must have gotten himself a girlfriend and she’s off barking orders

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    Mute Gaius Gracchus
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    Oct 5th 2013, 11:22 AM

    Apparently Capt Adebayo Flynn has run into a bit of bother, some people aren’t happy with his decision http://www.youtube.com/watch?v=kO12Ba5PwMA

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    Mute Suzanne Ní Dhúil
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    Oct 5th 2013, 11:41 AM

    Hilarious!!

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    Mute Jodi Steel
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    Oct 5th 2013, 1:44 PM

    That’s brilliant!

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    Mute Dreana O'Gorman
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    Oct 5th 2013, 2:45 PM

    Love it! :)

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    Mute Patricia Mc Cann
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    Oct 5th 2013, 3:32 PM

    Ha ha. He will be missed.

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    Mute Deirdre Mac Mahon
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    Oct 5th 2013, 11:17 AM

    ADEBAAAAAAAAAYOOOOOOO
    *sniff*
    well the Capt finally got me to open that twitter account.
    long life and prosper, Capt

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    Mute Nelly
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    Oct 5th 2013, 11:24 AM

    The capt had the ladies wrapped around his finger

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    Mute Niall H
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    Oct 5th 2013, 11:47 AM

    He’s some character. Pity there arent more like him on here. Its all cranky b******s now :)

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    Mute Abi Dennis
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    Oct 5th 2013, 12:11 PM

    I’m far more upset about Adebayo leaving than i should be…its worse than when Robbie left Take That!

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    Mute Abbi Cranky
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    Oct 5th 2013, 11:32 AM

    Oh Captain, my captain?

    (Standing on chair, fist over heart, sigh)

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    Mute Donal O Neil
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    Oct 5th 2013, 2:19 PM

    Captain for president , let’s vote him in ….

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    Mute tmwtbc
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    Oct 5th 2013, 11:41 AM

    Didn’t know the Captain had quit. Very funny guy and genuinely insightful.

    Good luck, Captain!

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    Mute Tigerisinthezoo
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    Oct 5th 2013, 12:21 PM

    After all the love from the women Adebayo is likely to come back in a new twitter life as Commander Adebayo Flynn!

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    Mute Michelle Mc Loughney
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    Oct 5th 2013, 12:05 PM

    Say it ain’t so captain. Say it ain’t so:( sobs.

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    Mute TheHassetthoff
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    Oct 5th 2013, 12:15 PM

    So long Captain, your posts are always top quality and good craic! Call back soon!

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    Mute Little Jim
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    Oct 5th 2013, 12:24 PM

    He’s not calling back, ever.
    He’s just too strong.
    *denial, tears, anger, acceptance, pride*

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    Mute Padraig Stapleton
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    Oct 5th 2013, 12:53 PM

    Farewell Adebayo, you legend! A great aul character…

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    Mute Marguerite Hoiby
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    Oct 5th 2013, 1:28 PM

    Why has he left?

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    Mute Mike Clinton
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    Oct 5th 2013, 3:08 PM

    So long Capt and your legacy will remain.
    Lelsie, lets ratchet it up a gear.

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    Mute Dave!
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    Oct 5th 2013, 4:27 PM

    You did good captain, consider yourself promoted to commandant.

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    Mute Tommy Hornblower
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    Oct 5th 2013, 1:46 PM

    I guess it’s up to Leslie Alan Rock to shepard the flock now…

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    Mute Beep Beep
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    Oct 5th 2013, 1:54 PM

    Oh god not Lesley Alan Moron, nobody wants his input

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    Mute Mike Clinton
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    Oct 5th 2013, 3:10 PM

    I disagree with you.
    I find Leslie’s posts quite good (and God knows I have had a few run in’s and don’t always agree)

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    Mute Leslie Alan Rock
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    Oct 5th 2013, 11:29 PM

    Thank you michael. ..onwards and side wards I shall lead you

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    Mute Mike Clinton
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    Oct 5th 2013, 11:37 PM

    Let’s Rock Leslie ;-). Take no prisoners.

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    Mute Dwickedchicken
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    Oct 5th 2013, 10:50 PM

    Personally I thought “capt” was an ass hole. :)

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    Mute Shane Hickey
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    Oct 11th 2013, 11:43 AM

    how do you quit an online forum? Surely it’s accessible from anywhere in the world (and on ISS)?

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