SMA Families in Northern Ireland Fighting for Nusinersen (Spinraza)
There are just four families in Northern Ireland who are affected by spinal muscular atrophy (SMA), three children have type 1 and the fourth has type 2.
The BBC reports that although nusinersen (Spinraza), the new treatment for SMA, has been approved by the Belfast Health Trust, the drug is yet to be licensed due to issues with administering the drug in the country.
Because the drug has not yet been licensed, the company who manufactures the product is willing to provide the drug for free to the affected families. But thus far the health trust has been unable to supply Spinraza to three of the four families because of technical difficulties finding somewhere for the drug to be administered.
The health trust has said that administering nusinersen to SMA children requires highly skilled doctors, nurses, access to theaters and other resources which it said “posed challenges to the health service.”
Understandably, the families involved are frustrated that their children have to wait for a potentially life-changing treatment and are pressuring the health trust via public appeals and through parliament.
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