Mar 20, 2023 · The majority of patients in LT-002 (70.4%, 57/81) never received add-on therapy (76.2% of the IV cohort, 50% of the investigational OAV101 IT ...

May 23, 2023 · All of the participants in LT001 received an intravenous (into the blood) injection of Zolgensma after the onset of SMA symptoms. In contrast, ...

Mar 16, 2021 · Two long-term studies of onasemnogene abeparvovec, LT-001 and LT-002, found that no serious adverse events (SAEs) led to study discontinuation ...

Apr 11, 2023 · Latest data from two Long-Term Follow-Up (LTFU) studies, LT-001 and LT-002, show the continued efficacy and durability of Zolgensma across a ...

May 22, 2023 · The two Long-Term Follow-Up studies are called LT-001 and LT-002, and are designed to assess the impact of Zolgensma for up to 15 years after ...

This MAD study will evaluate 3 dose levels of LT-002-158 once daily for consecutive 14 days. Thirty healthy volunteers will be enrolled into 3 cohorts and ...

Mar 23, 2021 · Data from LT-001 showed that Zolgensma promotes “sustained, durable responses for up to 5.6 years and for the LT-002 there is response that is ...

Apr 11, 2023 · LT-002 is a phase 4, 15-year ongoing follow-up safety and efficacy study of Zolgensma IV and investigational intrathecal (IT) OAV101 in ...

Mar 20, 2023 · One-time gene therapy Zolgensma from Novartis shows lasting benefits for SMA patients ; LT-002, IT Cohort, 18 ; Key: IV: Intravenous, IT: ...