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

Spinal muscular atrophy (SMA) ZOLGENSMA patient Lucy crawling on the floor

*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

Real ZOLGENSMA patient with SMA Type 2 Brady smiling

*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

Real ZOLGENSMA patient Maisie holding a cup and sitting with support in a high
chair

*As of December 2019. 

Steps to Treatment

What to know before, during and after infusion day

Steps to Treatment

STR1VE Trial

A Phase 3 clinical trial

STR1VE Trial