We need your support to keep TheyWorkForYou running and make sure people across the UK can continue to hold their elected representatives to account.

Donate to our crowdfunder

Spinal Muscular Atrophy Type 1: Treatment

Part of Question for Urgent Oral Answer — Health – in the Northern Ireland Assembly at 3:30 pm on 24th January 2017.

Alert me about debates like this

Photo of Michelle O'Neill Michelle O'Neill Sinn Féin 3:30 pm, 24th January 2017

This is a very sensitive issue, as these families are dealing with very difficult diagnoses of SMA and their children have very complex needs. I understand the concerns of those families involved and their request to have their children enrolled in the extended access programme of nusinersen, the potentially life-changing drug, to treat spinal muscular atrophy, and I am fully sympathetic to their concerns.

Clinicians in the Belfast Health and Social Care Trust made a clinical decision to use the extended access programme to provide this drug in an individual case to treat SMA. On this basis, the extension of this programme is a decision for the clinicians in the Belfast Trust. Like the Member, I am aware of the concerns of parents of children with SMA about the communication. My Department has raised these concerns with the trust, which in turn has assured me that urgent action will be taken to make contact with the families involved. I understand that direct contact will be made this Thursday with the families, offering a face-to-face meeting with the clinical team in the children's hospital.