Results 1 to 10 of about 1,998 (153)

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   +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   +3 more sources

Adeno-associated virus serotype 9 antibodies in patients screened for treatment with onasemnogene abeparvovec

Molecular Therapy: Methods & Clinical Development, 2021
Spinal muscular atrophy is a progressive, recessively inherited monogenic neurologic disease, the genetic root cause of which is the absence of a functional survival motor neuron 1 gene. Onasemnogene abeparvovec (formerly AVXS-101) is an adeno-associated
John W. Day, Richard S. Finkel, Eugenio Mercuri, Kathryn J. Swoboda, Melissa Menier, Rudolf van Olden, Sitra Tauscher-Wisniewski, Jerry R. Mendell   +7 more
doaj   +4 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   +4 more sources

Onasemnogene Abeparvovec Administration via Peripherally Inserted Central Catheter: A Case Report [PDF]

Children
Onasemnogene abeparvovec (OA) is the approved intravenous gene therapy for the treatment of spinal muscular atrophy (SMA). A functional copy of the human SMN1 gene was inserted into the target motor neuron cells via a viral vector, AAV9.
Inmaculada Pitarch Castellano, Eduardo López Briz, Eugenia Ibáñez Albert, Cristina Aguado Codina, Teresa Sevilla, José L. Poveda Andrés   +5 more
doaj   +4 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

Eculizumab for Thrombotic Microangiopathy Induced by Onasemnogene Abeparvovec in Spinal Muscular Atrophy [PDF]

Case Reports in Nephrology and Dialysis
Introduction: Onasemnogene abeparvovec is one of the three disease-modifying therapies available that can significantly improve the outcome of patients with 5q-spinal muscular atrophy.
Tanja Kersnik Levart, Nina Olas Kar, Chiara Močnik Pegan, Eva Vrščaj, Anja Troha Gergeli, Tanja Loboda, Damjan Osredkar   +6 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

Onasemnogene Abeparvovec in Early-Onset Spinal Muscular Atrophy: An Indian Experience [PDF]

Annals of Indian Academy of Neurology
Background and Objectives: Spinal muscular atrophy (SMA) is an inherited neuromuscular disorder with a grave prognosis. Gene replacement therapy has significantly altered the disease trajectory. This study presents real-world evidence of the efficacy and
Neelu Desai, Saheli Roy, Franzina Coutinho, Usha Kasar   +3 more
doaj   +2 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