Results 11 to 20 of about 1,998 (153)

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 McMillan, Laurent Servais, Michelle Farrar   +2 more
exaly   +7 more sources

Postmarketing adverse events associated with onasemnogene abeparvovec: a real-world pharmacovigilance study [PDF]

Orphanet Journal of Rare Diseases
Background Onasemnogene abeparvovec (OA) is an adeno-associated virus vector-based gene therapy indicated for the treatment of paediatric patients with spinal muscular atrophy(SMA) with biallelic mutations in the survival motor neuron 1 (SMN1) gene. This
Tianyu Chen, Qiying Chen, Jingfang Ye, Yuzhu Wu, Ting Liu, Yuezhen Zhang   +5 more
doaj   +3 more sources

Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec [PDF]

Molecular Therapy, 2023
Spinal muscular atrophy is an autosomal recessive disease resulting in motor neuron degeneration and progressive life-limiting motor deficits when untreated.
Ross Mangum, Nishant Tiwari
exaly   +3 more sources

Pilocytic astrocytoma in a child with spinal muscular atrophy treated with onasemnogene abeparvovec [PDF]

Molecular Therapy
Spinal muscular atrophy (SMA) is a severe neuromuscular disease, leading to progressive muscle weakness and potentially early mortality if untreated. Onasemnogene abeparvovec is a recombinant adeno-associated virus serotype 9 (rAAV9)-based gene therapy ...
Dorothea Holzwarth, Philipp John-Neek, Johannes Friese   +2 more
exaly   +3 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   +3 more sources

Efficacy and safety of onasemnogene abeparvovec for the treatment of patients with spinal muscular atrophy type 1: A systematic review with meta-analysis. [PDF]

PLoS ONE
BackgroundOnasemnogene abeparvovec has been approved for the treatment of spinal muscular atrophy 5q type 1 in several countries, which calls for an independent assessment of the evidence regarding efficacy and safety.ObjectiveConduct a meta-analysis to ...
Brígida Dias Fernandes, Bárbara Corrêa Krug, Fernanda D'Athayde Rodrigues, Hérica Núbia Cardoso Cirilo, Stéfani Sousa Borges, Ida Vanessa D Schwartz, Livia Fernandes Probst, Ivan Zimmermann   +7 more
doaj   +4 more sources

Clinical Trial and Postmarketing Safety of Onasemnogene Abeparvovec Therapy [PDF]

Drug Safety, 2021
This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy.We comprehensively assessed the safety of intravenous onasemnogene abeparvovec from preclinical studies, clinical studies, and postmarketing data.Single-dose toxicity ...
Day, John W, Mendell, Jerry R, Mercuri, Eugenio, Finkel, Richard S, Strauss, Kevin A, Kleyn, Aaron, Tauscher-Wisniewski, Sitra, Tukov, Francis Fonyuy, Reyna, Sandra P, Chand, Deepa H   +9 more
openaire   +6 more sources

Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury [PDF]

Pharmacology Research & Perspectives
Aims were to reveal types of onasemnogene abeparvovec‐xioi (OA)‐induced liver injury, their treatment patterns, utilization of healthcare, and treatment costs.
Andrej Belančić, Branislava Raičević, Ivana Stević, Dinko Vitezić, Slobodan M. Janković   +4 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

Spinal muscular atrophy - onasemnogene abeparvovec and other therapeutic options [PDF]

Farmacja Polska, 2020
Spinal muscular atrophy (SMA) is a neuromuscular disorder that results in the loss of motor neurons. SMA is caused by mutations in the SMN1 gene, leading to the decreased synthesis of the SMN protein, necessary for motor neuron survival. In the past, SMA
Aleksandra Alicja Majchrzak-Celińska, Anna Warych, Mikołaj Szoszkiewicz   +2 more
doaj   +3 more sources