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Family of 4-year-old girl devastated she might not get potentially life transforming drug Spinraza

The family of a four-year-old girl from York say they are devastated she might not qualify for a drug which could transform her life.

Matilda Jamieson's parents had been fighting to get her prescribed the drug Spinraza Credit: ITV News

Matilda Jamieson's parents had been fighting to get her prescribed the drug Spinraza, which can help youngsters with a rare muscle wasting condition.

Now the health guidance body NICE have updated their criteria, meaning some patients who need it, might no longer be eligible for a treatment.

Rachel Jamieson, Matilda's mum:

We're sorry if, as a result of any of our communications, people with SMA and their families were given the impression that access to nusinersen through our draft recommendations would be without restriction.

As is made clear in our draft guidance, because of the difficulties in being confident about the clinical evidence and the cost-effectiveness of nusinersen, it was necessary to agree access criteria for a period of further data collection to try and better understand how nusinersen affects people with SMA. The committee therefore agreed an MAA which could not only help answer these questions but also address the financial risk and challenges for implementation in the NHS.

We understand that the eligibility criteria for the MAA for nusinersen are disappointing for patients who may not meet the criteria. We are always very conscious of the responsibility we bear when making difficult decisions like these.

We can confirm that we have received an appeal against our draft guidance. The appeal specifically relates to the terms of the MAA under which nusinersen will be funded on the NHS while more data is collected.

We will now, in line with our process, review the appeal points raised to decide whether it can be considered by an appeal panel.

This has been a challenging appraisal and all parties - NHS England, NICE and Biogen - worked together extensively to reach an arrangement that allows access to nusinersen for most people with SMA type 1, 2,3 and pre-symptomatic patients.

– NICE spokersperson