Muscle Dystrophy and Association Scientific Congress 2024

March 3-6, 2024

Poster presentations:

Dabbous O, Khan F, Benguerba K, Reyna SP, Pickard S. Instruments for assessment of cognitive function in patients with SMA: a targeted literature review. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL. 

Finkel RS, Mathews K, Bernes S, et al. Outcomes in patients with SMA and one SMN2 gene copy: findings from the RESTORE registry. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL. 

Harmelink M, Servais L, Bass N, et al. Patterns of transient troponin I elevations in patients with SMA treated with onasemnogene abeparvovec: real-world data from the RESTORE registry. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL. 

McMillan H, Baranello G, Farrar MA, et al. Safety and efficacy of intravenous onasemnogene abeparvovec in pediatric patients with spinal muscular atrophy: interim findings from the phase 3 SMART study. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL.

Patel A, Toro W, Bourke S, et al. Treatment preferences in spinal muscular atrophy (SMA): a swing weighting study for caregivers of SMA type 1 and type 2 patients. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL.  

Servais L, Shieh PB, Goedeker N, et al. Real-world outcomes following onasemnogene abeparvovec  in patients with SMA and invasive ventilatory support: findings from the RESTORE registry. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL. 

Saute J, Muntadas J, Gurgel-Gianetti J, et al. Safety and tolerability of onasemnogene abeparvovec for patients with spinal muscular atrophy weighing ≤17 kg and ≤24 months old: phase 4 OFELIA study. Presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference, March 3-6, 2024, Orlando, FL.