START Long-Term Follow-Up (LTFU): ZOLGENSMA demonstrates durability of effect up to 7.5 years after treatment1

An ongoing, long-term follow-up study of patients from the START clinical trial (N=13)1,2

The long-term follow-up study of START began after the completion of the 24-month START trial and is planned to last 15 years. A total of 13 patients, 10 from the high-dose cohort and 3 from the low-dose cohort, are enrolled in the study.

The latest START long-term follow-up data

(May 2022 data cut)

A total of 10 out of 12 high-dose patients enrolled in an ongoing, observational, long-term follow-up of the START trial1

As of May 2022, the mean time since treatment was 6.9 years (range 6.4-7.5) and the mean age at last follow up was 7.1 years (range 6.6-7.9).1,a

aA 15-year, voluntary follow-up of patients in the START trial who received a high dose of ZOLGENSMA (n=10). In long-term follow-up, 6 patients had used another disease-modifying therapy.1

As of the May 2022 data cut1:

Event-free survival

100% (10/10) of patients

were alive and free of permanent ventilation1,a

ZOLGENSMA continues to provide durable efficacy up to 7.5 years after treatment1

The long-term follow-up study is an ongoing follow-up of the Phase 1 START trial.

All 10 patients from the START high-dose cohort who enrolled in the long-term follow-up were alive and free of permanent ventilation as of May 20221

Results from the Phase 1 START clinical trial and long-term follow-up including motor milestone achievements

aAs of the last visit prior to December 2019 data cut.

bBased on adverse event reporting death, withdrawal, or permanent ventilation. There were no in-person assessments between December 2019 and June 2020.

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