Apply now: feature your voice in our cell & gene therapy series
Cell and gene therapies are transforming rare disease treatment, offering new hope where options were once limited. But how do they work, and what choices exist for your community?
We're launching a special content series to help our 15k+ readers navigate this evolving landscape – and we need your voice!
In a recent study, Bistritzer et al. (2025) explored the effects of early treatment on preterm twins with Spinal Muscular Atrophy (SMA). The twins, born at 32 weeks and 2 days, were treated with risdiplam and later with onasemnogene abeparvovec at 35 and 43 weeks of gestational age, respectively. This case study is significant because there are no established guidelines for treating preterm infants with SMA, and the safety and efficacy of treatments in these cases are not well understood.
The findings from this study contribute to the limited data available on treatment options for preterm infants with SMA. The early intervention with these treatments in the twins suggests that presymptomatic and early treatment can be crucial for improving outcomes in SMA, even in preterm infants. This case underscores the importance of early detection and treatment in this population, providing valuable insights for future guidelines and interventions.
