Breakthrough in Prenatal SMA Therapy

Screening for spinal muscular atrophy (SMA) should be offered to all women who are considering pregnancy or are currently pregnant. SMA is an autosomal recessive disease characterized by degeneration of spinal cord motor neurons that leads to atrophy of skeletal muscle and overall weakness. Once identified, oral therapy may be started in the neonatal interval for those with the most severe phenotype (SMA-1). However, on Feb 19, 2025, a medical team piloted an investigational PRENATAL protocol as treatment starting in utero! While more data is needed, the results have been incredible. In this episode we will highlight this fascinating therapy which was "parent proposed". Listen in for details!