Patient profiles

Real patient stories.

See the results with one-time-only gene therapy. 

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 ~20 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. 

Louis 

Treated with ZOLGENSMA at ~2.5 months old, pictured here at ~4.5 years old*

To see Louis’ story, watch the video or download the PDF.

*As of Dec 2019. 

Louis’ Treatment Journey Timeline

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*As of December 2019. 

Adalyne 

Treated with ZOLGENSMA at ~2.5 months old, pictured here at ~4.5 years old* 

Adalyne participated in the START clinical study; after completing the study, Adalyne started another disease-modifying treatment for SMA beginning at ~2.5 years old. To see Adalyne’s story, watch the video or download the PDF.

*As of Dec 2019. 

Adalyne’s Treatment Journey Timeline

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*As of December 2019. 

Matteo  

Treated with ZOLGENSMA at ~1 month old, pictured here at ~4.5 years old* 

To see Matteo’s story, watch the video or download the PDF.

*As of Dec 2019. 

Matteo’s Treatment Journey Timeline

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*As of December 2019.