Breakthrough made in bid to treat incurable ‘floppy baby syndrome’

Researchers at the University of Edinburgh have found that infants suffering from a form of motor neurone disease could benefit from drugs that could improve their muscle strength.

Spinal muscular atrophy (SMA), also known as floppy baby syndrome, targets the body’s nerve cells – motor neurones – causing patients to have little or no control over their movements.

Children with SMA also suffer from unhealthy muscles, but tests carried out by the research team found the damage can be reversed.

Tom Gillingwater, professor of neuroanatomy at the University of Edinburgh, led the study.

He said: “By showing the important role that muscles play in this disease, we can now focus our efforts on trying to block the disease in all affected tissues of the body.”

The research team said SMA was incurable and often fatal, with 50 per cent of the most severe cases resulting in death by the age of two.

Children with SMA experience progressive muscle wastage, loss of mobility and motor function.

It affects one in 6,000 births and one in 40 people carry the genetic mutation that causes it.

It is hoped the findings will lead to new drugs and other treatments.