Results 11 to 20 of about 2,700 (197)

Safety of onasemnogene abeparvovec for patients with spinal muscular atrophy 8.5 kg or heavier in a Global Managed Access Program [PDF]

Pediatric Neurology, 2022
BACKGROUND: Spinal muscular atrophy is a rare, neurodegenerative disorder caused by biallelic deletions in the survival motor neuron (SMN1) gene. Onasemnogene abeparvovec is a one-time, intravenous gene replacement therapy designed to deliver the SMN1 ...
Chand, Deepa H, LaMarca, Nicole, Mitchell, Susan, Reyna, Sandra P, Sun, Rui, Sutter, Thao   +5 more
core   +6 more sources

Onasemnogene abeparvovec for the treatment of spinal muscular atrophy [PDF]

Expert Opinion on Biological Therapy, 2022
Gene therapy for spinal muscular atrophy (SMA) represents a significant milestone in the treatment of neurologic diseases. SMA is a neurodegenerative disease that results in motor neuron loss because of mutations of the survival motor neuron 1 gene, which directs survival motor neuron (SMN) protein production.
Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni, Laurent Servais   +5 more
exaly   +7 more sources

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. [PDF]

Lancet Reg Health Eur
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.
Gowda V, Atherton M, Murugan A, Servais L, Sheehan J, Standing E, Manzur A, Scoto M, Baranello G, Munot P, McCullagh G, Willis T, Tirupathi S, Horrocks I, Dhawan A, Eyre M, Vanegas M, Fernandez-Garcia MA, Wolfe A, Pinches L, Illingworth M, Main M, Abbott L, Smith H, Milton E, D'Urso S, Vijayakumar K, Marco SS, Warner S, Reading E, Douglas I, Muntoni F, Ong M, Majumdar A, Hughes I, Jungbluth H, Wraige E.   +36 more
europepmc   +5 more sources

An updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER)

Journal of Market Access & Health Policy, 2021
Background: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results.
Rebecca Dean, Ivar Jensen, Phil Cyr, Beckley Miller, Benit Maru, Douglas M. Sproule, Douglas E. Feltner, Thomas Wiesner, Daniel C. Malone, Matthias Bischof, Walter Toro, Omar Dabbous   +11 more
doaj   +2 more sources

Real-world outcomes of spinal muscular atrophy treatment with onasemnogene abeparvovec in Croatia: a comprehensive case series and literature review [PDF]

Frontiers in Medicine
IntroductionThe development of novel treatment options and the implementation of newborn screening programs have significantly transformed the landscape of care for patients with spinal muscular atrophy (SMA).
Ivan Lehman, Ivan Lehman, Matija Matošević, Lovro Lamot, Lovro Lamot, Branka Bunoza, Branka Bunoza   +6 more
doaj   +2 more sources

Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study [PDF]

Neurology and Therapy, 2023
Introduction Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier for the treatment of patients with spinal muscular atrophy (SMA). The JEWELFISH study (NCT03032172) was designed to assess the safety, tolerability, pharmacokinetics (PK ...
Claudia A. Chiriboga, Claudio Bruno, Tina Duong, Dirk Fischer, Eugenio Mercuri, Janbernd Kirschner, Anna Kostera-Pruszczyk, Birgit Jaber, Ksenija Gorni, Heidemarie Kletzl, Imogen Carruthers, Carmen Martin, Francis Warren, Renata S. Scalco, Kathryn R. Wagner, Francesco Muntoni, the JEWELFISH Study Group   +16 more
doaj   +5 more sources

Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy [PDF]

Journal of Hepatology, 2021
BACKGROUND & AIMS: Spinal muscular atrophy (SMA) is an autosomal recessive, childhood-onset motor neuron disease. Onasemnogene abeparvovec (OA) is a gene therapy designed to address SMA\u27s root cause.
Chand, Deepa, Kaufmann, Petra, Kleyn, Aaron, Kullak-Ublick, Gerd, McMillan, Hugh, Mohr, Franziska, Montgomery, Kyle, Sun, Rui, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy   +9 more
core   +4 more sources

Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy [PDF]

Annals of Clinical and Translational Neurology, 2022
Objective To provide a greater understanding of the tolerability, safety and clinical outcomes of onasemnogene abeparvovec in real‐world practice, in a broad population of infants with spinal muscular atrophy (SMA).
Arlene M. D'Silva, Sandra Holland, Didu Kariyawasam, Karen Herbert, Peter Barclay, Anita Cairns, Suzanna C. MacLennan, Monique M. Ryan, Hugo Sampaio, Nicholas Smith, Ian R. Woodcock, Eppie M. Yiu, Ian E. Alexander, Michelle A. Farrar   +13 more
doaj   +2 more sources

Paradoxical increase of neurofilaments in SMA patients treated with onasemnogene abeparvovec-xioi [PDF]

Frontiers in Neurology, 2023
Background/ObjectiveNeurofilament light chain (NfL) has been proposed as a biomarker reflecting disease severity and therapy response in children with spinal muscular atrophy type 1 and 2 (SMA1 and 2). The objective of this study was to examine how serum
Marina Flotats-Bastardas, Lisa Bitzan, Charlotte Grell, Kyriakos Martakis, Kyriakos Martakis, Benedikt Winter, Michael Zemlin, Claudia D. Wurster, Zeljko Uzelac, Claudia Weiß, Andreas Hahn   +10 more
doaj   +2 more sources

Real-World Outcomes in Patients with Spinal Muscular Atrophy Treated with Onasemnogene Abeparvovec Monotherapy: Findings from the RESTORE Registry. [PDF]

J Neuromuscul Dis
Motor neuron disease; Newborn screening; Spinal muscular atrophyEnfermedad de la neurona motora; Cribado neonatal; Atrofia muscular espinalMalaltia de la neurona motora; Cribratge neonatal; Atròfia muscular espinalBackground: Long-term, real-world ...
Servais L, Day JW, De Vivo DC, Kirschner J, Mercuri E, Muntoni F, Proud CM, Shieh PB, Tizzano EF, Quijano-Roy S, Desguerre I, Saito K, Faulkner E, Benguerba KM, Raju D, LaMarca N, Sun R, Anderson FA, Finkel RS.   +18 more
europepmc   +6 more sources