Efficacy of systemic therapy in breast cancer with CNS metastases: “Real-world” experience. Effect of CD4+PD-1+CXCR6+ T cells on the response of immune checkpoint inhibitor therapy in brain metastases ...

Spinraza is a medication used in children (including newborns) and adults to treat spinal muscular atrophy. Treatment begins with four loading doses, followed by one maintenance dose every 4 months.

The FDA approved the intrathecal gene therapy onasemnogene abeparvovec (Itvisma) to treat spinal muscular atrophy (SMA) in adults and pediatric patients 2 years and older with a confirmed mutation in ...