1. Real-world experience of gene therapy with onasemnogene-abeparvovec (Zolgensma®) for patients with SMA-type1 in UK. Issue 9 (12th August 2022) Authors: Gowda, Vasantha; Wraige, Elizabeth; Ong, Min; Atherton, Mark; Majumdar, Anirban; Marco, Silvia Sanchez; Hughes, Imelda; Mccullagh, Gary; Muntoni, Francesco; Jungbluth, Heinz Journal: Journal of neurology, neurosurgery and psychiatry Issue: Volume 93:Issue 9(2022) Page Start: e2 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Respiratory Trajectories in Type 2 and 3 Spinal Muscular Atrophy in the iSMAC Cohort Study. (26th January 2021) Authors: Trucco, Federica; Ridout, Deborah; Scoto, Mariacristina; Coratti, Giorgia; Main, Marion L.; Muni Lofra, Robert; Mayhew, Anna G.; Montes, Jacqueline; Pane, Marika; Sansone, Valeria; Albamonte, Emilio; D'Amico, Adele; Bertini, Enrico; Messina, Sonia; Bruno, Claudio; Parasuraman, Deepak; Childs, Ann... Journal: Neurology Issue: Volume 96:Number 4(2021) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗