Commercial patient stories

Real-world evidence of gene therapy

Lucy 

Treated with ZOLGENSMA at ~5 weeks old, pictured here at ~10 months old

Lucy was diagnosed with SMA at 11 days old through newborn screening in her state. To see Lucy’s story, watch the video or download the PDF.

Lucy’s Treatment Journey Timeline

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*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

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*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

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*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