The First Time.

No sign of symptoms.

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1. Doctors in the US have treated a fetus with a rapidly progressive congenital disease from inside the womb for the very first time. Instead of giving medicine to a child with severe neuromuscular complications after they are born, a new case study details how a prospective mother agreed to take the medicine for her developing fetus while she was still pregnant. A prenatal test had shown that her fetus held two genetic mutations indicating type 1 spinal muscular atrophy (SMA), which usually results in severe muscle weakness and breathing difficulties within six months of birth. Historically, most children with type 1 SMA die by their second birthday, usually from respiratory failure. However, the child in this study has so far made it to the age of two and a half years with no sign of symptoms. (Source: sciencealert.com, nejm.org/doi. The NEJM case study is here)