Joy for Rathcoole and Clondalkin families as miracle drug gets green light

by Padraig Conlon
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Local parents who never gave up hope on a brighter future for their children have received the most wonderful news.

This is because the HSE will fund the drug Spinraza for treatment of spinal muscular atrophy, a muscle-deteriorating disease which affects 26 children in Ireland.

Two of those young people, Glen McHugh Farrelly and Sam Bailey, live locally in Clondalkin and Rathcoole.

Their families, along with the families and friends of other children living with SMA, have campaigned tirelessly for the drug’s approval and this was acknowledged by Minister for Health Simon Harris on Tuesday morning as he commented on the HSE approving Spinraza

“I know how long the journey has been for families and I am very glad they will now have access to the drug,” he said.

One of those who made that long journey was Fiona Bailey, mother of nine year old Sam Bailey from Rathcoole.

“This means that Sam, and all the other children suffering from SMA in Ireland, will be able to begin their treatment of this amazing drug in the coming months,” she said following the announcement from the HSE.

“We are so grateful and unbelievably happy, a chance of a better life for our darling boy, Sam.

“Today we have been given the gift of hope.

“Sam is stronger than anyone I know, in his heart and in his soul.

“He is a fighter and now that Spinraza is a reality for us, the future looks brighter.”

Described as a “miracle drug” Spinraza is clinically effective and has been proven to work by slowing down the muscle wastage and in many cases allowing muscle strengthening.

The drug is administered by spinal injection every four months and is currently the only approved treatment available for patients of spinal muscular atrophy.

The result of a deficiency of protein called SMN, spinal muscular atrophy affects a child’s muscular development, the earlier the symptoms are noticed, the more severe the type.

A few months ago the HSE said it would again look at the possibility of approving the drug after Biogen dropped its asking price.

Originally when the request for availability was turned down, the HSE said the drug would cost about €600,000 in the first year to treat each child living with the disease in this country and €380,000 a year after that.

Anton Mannering Chairman of SMA Ireland, the representative organisation for people with SMA in this country, expressed disappointment that an age restriction will be put in place.

“It is simply not good enough that up to half the people with SMA will not have access to treatment,” he said.

“There are several young people with SMA who are in their late teens and a few who are in their early twenties.

“If treatment will be given to a girl who is 17 continuing til later in life, perhaps to her 40s or beyond, how can it be justifiable to not treat someone only a year or two older?

“It is intolerably cruel and discriminatory to inflict that type of injustice on a person suffering from a degenerative disease.

“Especially when the only difference between a treated an untreated individual might be which side of voting eligibility they might fall.”

“That is without considering those weaker older adults now starting to experience difficulty chewing and swallowing as this condition robs them of function.

“As I have said before, does somebody have to die before sense is seen?

“As a nation we must be better than this.

“We must do better than this.”

Also welcoming the news was Fianna Fáil TD for Dublin Mid-West, John Curran who has campaigned with the local families and raised the issue of funding for the drug in the Dail again last month.

“Spinal Muscular Atrophy is a serious degenerative neuromuscular condition that affects virtually every aspect of a person’s life,” he said.

“All these years that patients and their families have been enduring exhausting daily routines and lengthy stays in hospitals, they have also been tirelessly campaigning for access to a drug that can transform their quality of life.

“The HSE’s decision to finally reimburse Spinraza is a positive outcome for the lives of each of the 25 children most affected in Ireland.

“Their parents have been unrelenting in their fight to secure this medicine and I wish to foremost acknowledge their immense work.

“It was enlightening to play a small part in their campaign and I was pleased to emphasise in Dail Éireann on many occasions.

“Protest after protest, petition after petition, they have shown what can be achieved with determination.”

Local Independent Councillor Francis Timmons was also full of praise for the campaigning families.

‘’I am so delighted that Spinraza for people living with SMA will now be available for those who need it,” he said.

“Like Glen in Clondalkin and Sam in Rathcoole, awful that families had to fight so hard but delighted that common sense has won out.

“There can be no cost on a life we all deserve the best chances in life.”

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