World Muscle Society Congress 2023

October 3-7, 2023

Poster presentations:

McGill B, Thach C, Tauscher-Wisniewski S, Macek TA. Post-hoc analysis of compound motor action potential from clinical trials of intravenous onasemnogene abeparvovec for spinal muscular atrophy. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Mendell JR, Wigderson M, Alecu I, Yang L, Mehl L, Connolly AM. Long-term follow-up of onasemnogene abeparvovec gene therapy in symptomatic patients with spinal muscular atrophy type 1. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Darras BT, Mercuri E, Strauss KA, et al. Intravenous and intrathecal onasemnogene abeparvovec gene therapy in symptomatic and presymptomatic spinal muscular atrophy: long-term follow-up study. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Macek TA, Wen S, O’Brien E, Alecu I, Tauscher-Wisniewski S. Post-hoc analyses of prednisolone use and hepatotoxicity in clinical trials of intravenous onasemnogene abeparvovec. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Dabbous O, LaMarca N, Toro W, et al. Real-world outcomes of disease-modifying treatment for patients with spinal muscular atrophy: findings from a global retrospective chart review. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Servais L, Benguerba K, Gehani M, et al. Outcomes in patients with spinal muscular atrophy and four or more SMN2 copies treated with onasemnogene abeparvovec: findings from RESTORE registry. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Finkel RS, Dabbous O, Benguerba K, Mumneh N, Raju D, Servais L. Real-world data for patients with gestational age ≤35 weeks at birth treated with onasemnogene abeparvovec: results from the RESTORE registry. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.

Toro W, Reyna SP, Ritter S, Patel A, Mumneh N, Dabbous O. Early intervention and speed-to-effect in spinal muscular atrophy type 1 following onasemnogene abeparvovec gene replacement therapy. Presented at the 28th Annual Congress of the World Muscle Society, October 3-7, 2023, Charleston, SC, USA.