Patient profiles
Commercial patient stories
Real-world evidence of gene therapy
Payne
Treated with ZOLGENSMA at ~6 weeks old, pictured here at ~1.5 years old
Payne’s SMA was first identified through newborn screening. His SMA diagnosis was confirmed through genetic testing, which found he has 4 copies of SMN2.
Payne’s Treatment Journey Timeline
*As of December 2019.
Lucy
Treated with ZOLGENSMA at ~5 weeks old, pictured here at ~10 months old
Lucy was diagnosed with SMA at 11 days old, after SMA was identified through newborn screening in her state. To see Lucy’s story, watch the video or download the PDF.
Lucy’s Treatment Journey Timeline
*As of December 2019.
Brady
Treated with ZOLGENSMA at 14 months old, pictured here at ~2 years old
Brady was diagnosed with SMA Type 2 at 13 months old. See his journey from diagnosis to treatment at 14 months old–watch the video or download the PDF.
Brady’s Treatment Journey Timeline
*As of December 2019.
Maisie
Treated with ZOLGENSMA at ~20 months old, pictured here at ~2 years old
Maisie also received another disease-modifying treatment for SMA from ~6 months to ~18 months. To see Maisie’s story, watch the video or download the PDF.
Maisie’s Treatment Journey Timeline
*As of December 2019.