Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
Titel:
Efficacy and safety of onasemnogene abeparvovec in children with spinal muscular atrophy type 1: real-world evidence from 6 infusion centres in the United Kingdom
Auteur:
Gowda, Vasantha Atherton, Mark Murugan, Archana Servais, Laurent Sheehan, Jennie Standing, Emma Manzur, Adnan Scoto, Mariacristina Baranello, Giovanni Munot, Pinki McCullagh, Gary Willis, Tracey Tirupathi, Sandya Horrocks, Iain Dhawan, Anil Eyre, Michael Vanegas, Maria Fernandez-Garcia, Miguel A. Wolfe, Amy Pinches, Laura Illingworth, Marjorie Main, Marion Abbott, Lianne Smith, Hayley Milton, Emily D’Urso, Sarah Vijayakumar, Kayal Marco, Silvia Sanchez Warner, Sinead Reading, Emily Douglas, Isobel Muntoni, Francesco Ong, Min Majumdar, Anirban Hughes, Imelda Jungbluth, Heinz Wraige, Elizabeth