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Page last updated: 23rd May 2023

Data presented at the 2023 Muscular Dystrophy Association Conference, revealed that children treated with Zolgensma maintained or achieved additional milestones up to 7.5 years after administration of the drug.

Given the time scales involved in obtaining and analysing the trial results, the presented information is accurate as of the 23rd May 2022.

Results from both studies show the continued effectiveness and durability of Zolgensma, which continues to display a favourable benefit-to-risk profile.

All of the participants in LT001 received an intravenous (into the blood) injection of Zolgensma after the onset of SMA symptoms.

In contrast, LT-002 includes participants who received Zolgensma either before or after symptoms started, and includes people that received either intravenous or intrathecal (into the spinal fluid) doses of the therapy. The intrathecal treatment approach is now being referred to as “OAV101 IT” or “OAV101”.

Importantly, people in both trials have all maintained previously achieved motor milestones. Furthermore, in LT-001, three additional participants (30%) also achieved the key milestone of “standing with assistance.” In LT-002, 48 out of 77 (62.3%) participants either achieved a new motor milestone or had already achieved all motor milestones in the study in which they were initially enrolled.

Together, these results show that Zolgensma continues to be effective, safe and durable in a range of people with SMA.

Further Information

Novartis Press Release >

LT-001 Clinical Trials Page >

LT-002 Clinical Trials Page >

Previous Updates

27th March:

New data from the START long-term follow-up study (LT001) continued to demonstrate the durability of a single, one-time dose of Zolgensma in patients now up to five years after their treatment. Some of these Type 1 patients were now more than five years of age.

All children in this study who received Zolgensma were alive, free of permanent ventilation and continued to maintain developmental milestones. This included two children who achieved the new milestone of standing with assistance during the long-term follow-up period.