Results 11 to 20 of about 3,107 (205)

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   +7 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   +3 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

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

Eur J Health Econ
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 with SMA ...
van der Schans S, Velikanova R, Weidlich D, Howells R, Patel A, Bischof M, Postma MJ, Boersma C.   +7 more
europepmc   +4 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   +3 more sources

Gene Therapy for Spinal Muscular Atrophy (SMA): A Review of Current Challenges and Safety Considerations for Onasemnogene Abeparvovec (Zolgensma). [PDF]

Cureus, 2023
Spinal Muscular Atrophy (SMA) is a genetic disease that causes weakness and wasting in the voluntary muscles of infants and children. SMA has been the leading inherited cause of infant death.
Ogbonmide T, Rathore R, Rangrej SB, Hutchinson S, Lewis M, Ojilere S, Carvalho V, Kelly I.   +7 more
europepmc   +4 more sources

Experience of using gene replacement therapy with Zolgensma® (onasemnogene abeparvovec) in real clinical practice in Russia [PDF]

Нервно-мышечные болезни, 2022
Objective: to analyze the safety and evaluate the effectiveness of therapy with onasemnogene abeparvovec in patients with spinal muscular atrophy in real clinical practice based on the experience of using the drug in the neuromuscular center of Research ...
S. B. Artemyeva, Yu. O. Papina, O. A. Shidlovskaya, A. V. Monakhova, D. V. Vlodavets   +4 more
doaj   +5 more sources

Real-world multidisciplinary outcomes of onasemnogene abeparvovec monotherapy in patients with spinal muscular atrophy type 1: experience of the French cohort in the first three years of treatment. [PDF]

Orphanet J Rare Dis
Spinal muscular atrophy type 1 (SMA1) is the most severe and early form of SMA, a genetic disease with motor neuron degeneration. Onasemnogene abeparvovec gene transfer therapy (GT) has changed the natural history of SMA1, but real-world data are scarce.
Desguerre I, Barrois R, Audic F, Barnerias C, Chabrol B, Davion JB, Durigneux J, Espil-Taris C, Gomez-Garcia de la Banda M, Guichard M, Isapof A, Nougues MC, Laugel V, Le Goff L, Mercier S, Pervillé A, Richelme C, Thibaud M, Sarret C, Schweitzer C, Testard H, Trommsdorff V, Vanhulle C, Walther-Louvier U, Altuzarra C, Chouchane M, Ropars J, Quijano-Roy S, Cances C.   +28 more
europepmc   +4 more sources

Onasemnogene abeparvovec for spinal muscular atrophy. [PDF]

Aust Prescr, 2022
J Mendell, S Al-Zaidy, R Shell, W Arnold, L Rodino-Klapac, T Prior, J Day, R Finkel, C Chiriboga, A Connolly, T Crawford, B Darras, E Mercuri, F Muntoni, G Baranello, R Masson, O Boespflug-Tanguy, C Bruno, K Strauss, M Farrar, F Muntoni, K Saito, J Mendell, L Servais, K Strauss, M Farrar, F Muntoni, K Saito, J Mendell, L Servais   +29 more
europepmc   +5 more sources

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy: The Phase 3b SMART Study. [PDF]

Neurology
Background and Objectives Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing 13–17, and >17–21 kg), administered onasemnogene abeparvovec, and followed for 52 weeks ...
McMillan HJ, Baranello G, Farrar MA, Zaidman CM, Moreno T, De Waele L, Jong YJ, Laugel V, Quijano-Roy S, Mercuri E, Chien YH, Straub V, Darras BT, Seibert J, Bernardo Escudero R, Alecu I, Freischläger F, Muntoni F, SMART Study Group.   +18 more
europepmc   +3 more sources