News Story

A new treatment for babies diagnosed with a rare genetic condition has been approved for use by the NHS in Scotland.

The Scottish Medicines Consortium (SMC), the body which decides what treatments can be offered on the NHS, has approved a new drug for treating spinal muscular atrophy (SMA) in babies even before they display symptoms.

The drug nusinersen which is also known as Spinraza was approved for use on the NHS in the rest of the UK in June this year.

Now the SMC has recommended it can also be used for babies and children in Scotland who are diagnosed with the condition.

SMC chair Dr Rob Peel described SMA as being a “complex condition for patients and their families”.

He added that “nusinersen is another treatment option for use, before symptoms develop, which may be better suited for some patients”.

The decision to make the drug which is given as a regular injection into spinal fluid   available on the NHS comes after Scotland became the first part of the UK to routinely screen newborn infants for SMA in March this year.

The disease is a rare genetic condition that causes progressive muscle weakness and wasting, which can affect movement, breathing and swallowing.

While symptoms can develop rapidly in infancy, medics have found that treating babies before their symptoms can significantly improve outcomes.

Portia Thorman, from the charity Spinal Muscular Atrophy UK, said approving nusinersen for use by the NHS was a “welcome and meaningful decision for the SMA community in Scotland”.

With two other treatments also available prior to symptoms starting, she said that “families with babies identified through newborn screening can make the best treatment choice for their individual situation, with the support of their specialist clinicians”.