Doctors have managed to treat a deadly motor neuron disease in a baby who was still in the womb. The details have been published in The New England Journal of Medicine. It marks the first time that the condition, called spinal muscular atrophy (SMA) has been tackled before birth. SMA is a genetic disorder that targets the motor neurons, limiting movement and weakening muscles over time. It is an extremely rare disease, occurring in just one out of every 10,000 live births. The child in this case had SMA type 1, the most common form of the disorder. In the months since her birth has shown no signs of abnormal muscle development. Although she may have to take risidiplam for the rest of her life, into adulthood while doctors closely monitor her for any changes in muscle development, the baby had shown no sign of the rare genetic disorder, nearly three years after being born. The doctors found that the infant had higher levels of SMN protein and less nerve damage than other babies born with SMAtype 1.
