Vasantha Gowda, Mark Atherton, Archana Murugan, Laurent Servais, Jennie Sheehan, Emma Standing, Adnan Manzur, Mariacristina Scoto, Giovanni Baranello, Pinki Munot, Gary McCullagh, Tracey Willis, Sandya Tirupathi, Iain Horrocks, Anil Dhawan, Michael Eyre, Maria Vanegas, Miguel A Fernandez-Garcia, Amy Wolfe, Laura Pinches, Marjorie Illingworth, Marion Main, Lianne Abbott, Hayley Smith, Emily Milton, Sarah D'Urso, Kayal Vijayakumar, Silvia Sanchez Marco, Sinead Warner, Emily Reading, Isobel Douglas, Francesco Muntoni, Min Ong, Anirban Majumdar, Imelda Hughes, Heinz Jungbluth, Elizabeth Wraige
BACKGROUND: Real-world data on the efficacy and safety of onasemnogene abeparvovec (OA) in spinal muscular atrophy (SMA) are needed, especially to overcome uncertainties around its use in older and heavier children. This study evaluated the efficacy and safety of OA in patients with SMA type 1 in the UK, including patients ≥2 years old and weighing ≥13.5 kg. METHODS: This observational cohort study used data from patients with genetically confirmed SMA type 1 treated with OA between May 2021 and January 2023, at 6 infusion centres in the United Kingdom...
February 2024: The Lancet regional health. Europe