Results 31 to 40 of about 2,700 (197)

Changes in electrophysiological findings of spinal muscular atrophy type I after the administration of nusinersen and onasemnogene abeparvovec: two case reports. [PDF]

BMC Neurol, 2023
Mizuno T, Kanouchi T, Tamura Y, Hirata K, Emoto R, Suzuki T, Kashimada K, Morio T.   +7 more
europepmc   +3 more sources

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study. [PDF]

Neurology
McMillan HJ, Baranello G, Farrar MA, Zaidman CM, Moreno T, De Waele L, Jong YJ, Laugel V, Quijano-Roy S, Mercuri E, Chien YH, Straub V, Darras BT, Seibert J, Bernardo Escudero R, Alecu I, Freischläger F, Muntoni F, SMART Study Group.   +18 more
europepmc   +2 more sources

Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands. [PDF]

Eur J Health Econ
van der Schans S, Velikanova R, Weidlich D, Howells R, Patel A, Bischof M, Postma MJ, Boersma C.   +7 more
europepmc   +3 more sources

Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial. [PDF]

Nat Med
Kwon JM, Munell F, Le Goff L, Yuge K, Kato T, Cances C, De Waele L, Woodcock IR, Mercuri EM, Proud CM, Darras BT, Hayes LH, Oskoui M, Visootsak J, Williams G, Ilić A, Yang L, van der Pol WL.   +17 more
europepmc   +3 more sources

Summary of Research: Risdiplam Treatment Following Onasemnogene Abeparvovec in Individuals with Spinal Muscular Atrophy: A Multicenter Case Series. [PDF]

Adv Ther
Svoboda MD, Kuntz N, Leon-Astudillo C, Byrne BJ, Krueger J, Kwon JM, Sieburg C, Castro D.   +7 more
europepmc   +3 more sources

Safety and efficacy of intravenous onasemnogene abeparvovec gene therapy in patients with spinal muscular atrophy type 1: interim analysis from LT-001, a long-term follow-up study of patients from the START study. [PDF]

EClinicalMedicine
Waldrop MA, Escudero RB, Yang L, Camarata RA, Branic EC, Mehl L, Ilić A, Connolly AM.   +7 more
europepmc   +2 more sources

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment. [PDF]

Orphanet J Rare Dis
Desguerre I, Barrois R, Audic F, Barnerias C, Chabrol B, Davion JB, Durigneux J, Espil-Taris C, Gomez-Garcia de la Banda M, Guichard M, Isapof A, Nougues MC, Laugel V, Le Goff L, Mercier S, Pervillé A, Richelme C, Thibaud M, Sarret C, Schweitzer C, Testard H, Trommsdorff V, Vanhulle C, Walther-Louvier U, Altuzarra C, Chouchane M, Ropars J, Quijano-Roy S, Cances C.   +28 more
europepmc   +3 more sources

Judicialization of Zolgensma in the Ministry of Health: costs and clinical profile of patients [PDF]

Revista de Saúde Pública
OBJECTIVE To investigate the costs and profile of patients who have filed a lawsuit against the Ministry of Health for the treatment of spinal muscular atrophy (SMA) with the onasemnogene abeparvovec (Zolgensma®).
Ana Katheryne Miranda Kretzschmar, Ellen Teixeira, Dayani Galato, Everton Nunes da Silva   +3 more
doaj   +4 more sources

Respiratory outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy: national real-world cohort study. [PDF]

Eur J Pediatr
Lavie M, Rochman M, Armoni Domany K, Golan Tripto I, Be'er M, Besor O, Sagi L, Aharoni S, Ginsberg M, Noyman I, Levine H.   +10 more
europepmc   +3 more sources

Treating Presymptomatic Spinal Muscular Atrophy Patients with Onasemnogene Abeparvovec in Italy: The Role of the National Health System and Drug Supply. Comment on Zaidman et al. Newborn Screening for Spinal Muscular Atrophy: Variations in Practice and Early Management of Infants with Spinal Muscular Atrophy in the United States. <i>Int. J. Neonatal Screen.</i> 2024, <i>10</i>, 58. [PDF]

Int J Neonatal Screen
Masson R, Gaballo S, Caravita R, Parravicini S.   +3 more
europepmc   +3 more sources