Results 1 to 10 of about 3,107 (205)

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   +7 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   +5 more sources

Comprehensive analysis of adverse events associated with onasemnogene abeparvovec (Zolgensma) in spinal muscular atrophy patients: insights from FAERS database [PDF]

Frontiers in Pharmacology
Onasemnogene Abeparvovec (Zolgensma) is a gene therapy for the treatment of Spinal Muscular Atrophy (SMA) with improved motor neuron function and the potential for a singular treatment.
Wenwen Zhang, Yizhen Yin, Dan Yang, Mengyuan Liu, Caixia Ye, Ruiling Yan, Ruiman Li   +6 more
doaj   +6 more sources

Risdiplam treatment following onasemnogene abeparvovec in individuals with spinal muscular atrophy: a multicenter case series [PDF]

BMC Neurology
Background Spinal muscular atrophy (SMA) is caused by deletions or mutations in the survival of motor neuron (SMN) 1 gene resulting in progressive motor function loss, and additional disease-related complications, including dysphagia and respiratory ...
Melissa D. Svoboda, Nancy Kuntz, Carmen Leon-Astudillo, Barry J. Byrne, Jena Krueger, Jennifer M. Kwon, Cory Sieburg, Diana Castro   +7 more
doaj   +5 more sources

Single-Dose Intrathecal Dorsal Root Ganglia Toxicity of Onasemnogene Abeparvovec in Cynomolgus Monkeys. [PDF]

Hum Gene Ther, 2022
Intravenous onasemnogene abeparvovec is approved for the treatment of spinal muscular atrophy in children \u3c 2 years. For later-onset patients, intrathecal onasemnogene abeparvovec may be advantageous over intravenous administration.
Tukov FF, Mansfield K, Milton M, Meseck E, Penraat K, Chand D, Hartmann A.   +6 more
europepmc   +5 more sources

Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy [PDF]

Pharmaceutics, 2023
Spinal muscular atrophy (SMA) is a devastating neuromuscular disorder caused by mutations in the survival of motor neuron 1 (SMN1) gene, which leads to a reduced level in the SMN protein within cells.
Charlotte A. René, Robin J. Parks
doaj   +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   +5 more sources

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

Drug Saf, 2021
INTRODUCTION: This is the first description of safety data for intravenous onasemnogene abeparvovec, the only approved systemically administered gene-replacement therapy for spinal muscular atrophy.
Day JW, Mendell JR, Mercuri E, Finkel RS, Strauss KA, Kleyn A, Tauscher-Wisniewski S, Tukov FF, Reyna SP, Chand DH.   +9 more
europepmc   +9 more sources

Onasemnogene‐abeparvovec administration to premature infants with spinal muscular atrophy [PDF]

Annals of Clinical and Translational Neurology
Twin girls born at 30 weeks' gestation with spinal muscular atrophy (SMA) received onsasemnogene‐abeparvovec (OA) at 3.5 weeks of life. They had no treatment‐related adverse events, normal acquisition of motor milestones, and normal neurological ...
Stephen M. Brown, Aparna S. Ajjarapu, Divya Ramachandra, Laura Blasco‐Pérez, Mar Costa‐Roger, Eduardo F. Tizzano, Charlotte J. Sumner, Katherine D. Mathews   +7 more
doaj   +6 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