CureSMA Annual SMA Conference 2023

June 28-June 30, 2023

Poster presentations:

McGrattan KE, Shell RD, Hurst-Davis R, et al. Bulbar function in children with two or three SMN2 Copies who received onasemnogene abeparvovec presymptomatically for spinal muscular atrophy. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Mendell JR, Wigderson M, Alecu I, et al. Long-term follow-up of onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Connolly A, 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 Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Dabbous O, Shell R, Ritter S, et al. Health outcomes impacting quality of life in spinal muscular atrophy following onasemnogene abeparvovec gene replacement therapy. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Proud C, Mercuri E, Finkel R, et al. Combination disease-modifying treatment (DMT) in spinal muscular atrophy (SMA): A proposed classification for future analyses. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, 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 Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Servais L, Day JW, De Vivo DC, et al. Real-world outcomes in patients with spinal muscular atrophy treated with onasemnogene abeparvovec as monotherapy: findings from the RESTORE registry. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.

Toro W, Patel A, Dabbous O. Burden of spinal muscular atrophy type 3 on caregivers in the United States: results of a global survey. Presented at the Cure SMA 2023 Annual SMA Research & Clinical Care Meeting, June 28–30, 2023, Orlando, FL, USA.