Relief for families of Clondalkin and Rathcoole boys as “miracle drug” Spinraza approved by HSE

by Padraig Conlon

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 have been campaigning tirelessly for the drug’s approval, which Minister for Health Simon Harris announced earlier as he paid tribute to all the campaigners.

“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.

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 children living with the disease in this country and €380,000 a year after that.

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