Results 51 to 60 of about 152 (86)

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

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   +1 more source

Real-world effectiveness and economic evaluations of EMA-approved orphan medicines for spinal muscular atrophy: a systematic literature review [PDF]

Pharmacia
Background: Spinal muscular atrophy (SMA) is a rare neuromuscular disorder associated with a substantial clinical burden, high supportive-care needs, and a major economic impact.
Neli Boseva-Stoyanova, Teodora Chamova, Guenka Petrova, Alexandra Savova   +3 more
doaj   +3 more sources

Disease Modifying Therapies for the Management of Children with Spinal Muscular Atrophy (5q SMA): An Update on the Emerging Evidence

Drug Design, Development and Therapy, 2022
Helgi Thor Hjartarson,1 Kristofer Nathorst-Böös,1 Thomas Sejersen1,2 1Department of Neuropediatrics, Astrid Lindgren Children´s Hospital, Karolinska University Hospital, Stockholm, Sweden; 2Department of Women’s and Children’s Health, Karolinska ...
Hjartarson HT, Nathorst-Böös K, Sejersen T   +2 more
doaj  

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

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   +1 more source

A consensus survey of neurologists and clinical geneticists on spinal muscular atrophy treatment in Singapore

Annals, Academy of Medicine, Singapore
Just a decade ago, spinal muscular atrophy (SMA) was considered a debilitating, progressive neuromuscular disease that inevitably led to chronic disability and a shortened lifespan.
Jocelyn Yi Xiu Lim, Furene Sijia Wang, Simon Robert Ling, Stacey Kiat Hong Tay   +3 more
doaj   +1 more source

Pioneering SMA therapies for all types: survival gains, cost dynamics, and performance-based agreements

Cost Effectiveness and Resource Allocation
Background The purpose of this study was to assess the impact of survival improvements and performance-based managed entry agreements (PBMEAs) on the cost implications of introducing innovative spinal muscular atrophy (SMA) treatments, nusinersen ...
Ahmed Al-jedai, Hajer Al-Mudaiheem, AlJohara AlSakran, Fahad A. Bashiri, Fouad Ghamdi, Mohammad A. Almuhaizea, Abdulaziz AlSamman, Nancy Awad, Rita Ojeil   +8 more
doaj   +1 more source

Experience and Perspectives in the US on the Evolving Treatment Landscape in Spinal Muscular Atrophy

International Journal of General Medicine, 2022
Leigh Ramos-Platt,1 Lauren Elman,2 Perry B Shieh3 1Department of Pediatrics, Keck School of Medicine, University of Southern California and Children’s Hospital of Los Angeles, Los Angeles, CA, USA; 2Department of Neurology, University of Pennsylvania ...
Ramos-Platt L, Elman L, Shieh PB
doaj  

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

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   +1 more source

Costs of Treating Onasemnogene Abeparvovec‐Xioi‐Induced Liver Injury

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   +1 more source

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

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   +1 more source