Prenatal Treatment for Spinal Muscular Atrophy

Clinical trials demonstrated that spinal muscular atrophy (SMA) can be treated prenatally using risdiplam, an RNA splicing modifier. The study showed that newborns destined to develop SMA could begin treatment at birth safely and effectively. This represents the first prenatal therapy for the genetic condition, potentially preventing muscle degeneration before symptoms appear. The breakthrough offers hope for early intervention in genetic diseases and may establish new protocols for fetal medicine