Realising milestones with gene therapy for SMA
Novartis Gene Therapies' Dr Sitra Tauscher-Wisniewski shares what shaped the success of one of the few commercially-available gene therapies.
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Novartis Gene Therapies' Dr Sitra Tauscher-Wisniewski shares what shaped the success of one of the few commercially-available gene therapies.
Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusionAll...
20.03.2023 - Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusionAll children (100%) in the presymptomatic intravenous cohort of LT-002 maintained or achieved all ...
Children in LT-001 treated after SMA symptom onset maintained or achieved additional milestones up to 7.5 years post one-time intravenous infusionAll children (100%) in the presymptomatic intravenous cohort
BASEL (dpa-AFX) - Novartis AG (NVS) Monday presented new long-term data of Zolgensma (onasemnogene abeparvovec), the only approved one-time gene therapy for the treatment of spinal muscular atrophy