Results 11 to 20 of about 2,067 (154)

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, Laurent Servais, Michelle A Farrar   +2 more
exaly   +7 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, Brent Kuzmiski   +2 more
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, Johannes Friese, Philipp John-Neek   +2 more
exaly   +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

Combination disease‐modifying treatment in spinal muscular atrophy: A proposed classification [PDF]

Annals of Clinical and Translational Neurology, 2023
We sought to devise a rational, systematic approach for defining/grouping survival motor neuron‐targeted disease‐modifying treatment (DMT) scenarios.
Crystal M. Proud, Eugenio Mercuri, Richard S. Finkel, Janbernd Kirschner, Darryl C. De Vivo, Francesco Muntoni, Kayoko Saito, Eduardo F. Tizzano, Isabelle Desguerre, Susana Quijano‐Roy, Kamal Benguerba, Dheeraj Raju, Eric Faulkner, Laurent Servais   +13 more
doaj   +5 more sources

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

European Journal of Health Economics
BACKGROUND: Spinal muscular atrophy (SMA) is a rare genetic disease resulting in loss of motor function and, in severe cases (e.g., SMA type 1), infantile death. While treatments like nusinersen and onasemnogene abeparvovec improve prognosis for patients
Cornelis Boersma, Maarten J. Postma, Simon van der Schans   +2 more
exaly   +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   +3 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

Early Intervention and Speed-to-Effect in Spinal Muscular Atrophy Type 1 Following Onasemnogene Abeparvovec Gene Replacement Therapy: Results of aPost - Hoc Analysis of Pooled Clinical Study Data [PDF]

Neurology and Therapy
Introduction Studies suggest that early intervention with disease-modifying treatment for spinal muscular atrophy (SMA) might provide the best opportunity for optimal outcomes.
Walter Toro, Sandra P. Reyna, Shannon Ritter, Anish Patel, Nayla Mumneh, Omar Dabbous   +5 more
doaj   +2 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