Results 81 to 90 of about 1,998 (153)

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

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  

Pharmacokinetics of therapies approved for spinal muscular atrophy: A narrative review of current evidence

Journal of International Medical Research
Spinal muscular atrophy is a severe neuromuscular disorder caused by mutations in the survival motor neuron 1 gene, leading to progressive motor neuron degeneration.
Eda Kübra Sel, Elvira Meni Maria Gkrinia, Rossana Roncato, Dinko Vitezić, Slobodan Janković, Andrej Belančić,   +6 more
doaj   +1 more source

Spinal muscular atrophy in the disease-modifying therapy era: successes, limitations and future directions

Frontiers in Molecular Medicine
Spinal Muscular Atrophy (SMA) is a rare and debilitating neurodegenerative disease characterized by the progressive loss of motor neurons in the spinal cord, leading to muscle weakness, respiratory failure, and premature mortality.
Madison M. Sexton, Emma W. Crow, Congyue Annie Peng   +2 more
doaj   +1 more source

Onasemnogene abeparvovec (Zolgensma®) for the treatment of spinal muscular atrophy: A Protocol for Systematic Review

This project contains the protocol, search strategies, study selection materials, data extraction forms, risk-of-bias assessments, GRADE evidence profiles, PRISMA flow diagram, and manuscript files for a rapid systematic review and health technology ...
LINA SOFIA MORON DUARTE, Kelly Chacon, Ana Maria Rojas, Maria Paula Gutierrez, Adriana Rodriguez   +4 more
core   +1 more source

Scoliosis in Spinal Muscular Atrophy: Current Approach Toward Management

Indian Spine Journal
Spinal muscular atrophy (SMA) is a hereditary neuromuscular disorder characterized by degeneration of anterior horn cells in the spinal cord, resulting in progressive muscular weakness.
Saumyajit Basu, Ayon Ghosh
doaj   +1 more source

ID182 Eficácia e efetividade do nusinersena, risdiplam e onasemnogeno abeparvoveque para o tratamento de atrofia muscular espinhal (AME) 5q tipos I e II em relação ao número de cópias do gene SMN2

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução Atrofia muscular espinhal (AME 5q) é caracterizada pela degeneração dos neurônios motores inferiores que, consequentemente, leva à paralisia com atrofia muscular associada a mutações bialélicas do gene SMN1.
Roberto Lúcio Muniz Júnior, Álex Brunno do Nascimento Martins, Bárbara Rodrigues Alvernaz dos Santos, Ludmila Peres Gargano, Marcus Carvalho Borin, Marcus Carvalho Borin, Francisco de Assis Acurcio, Juliana Alvares-Teodoro, Augusto Afonso Guerra Júnior   +7 more
doaj   +1 more source

PE-068 Impacto econômico nas famílias de crianças com atrofia muscular espinhal tipo 1 no Brasil: um estudo transversal

Jornal de Assistência Farmacêutica e Farmacoeconomia
Introdução: A atrofia muscular espinhal (AME) é uma doença neuromuscular rara, progressiva e debilitante. Apesar dos subsídios para medicamentos, a AME tipo I pode gerar despesas catastróficas para as famílias dos pacientes afetados [1–3]. Objetivo: Este
André Soares Motta-Santos, Kenya Noronha, Carla Barros Reis, Daniela Almeida Freitas, Lélia Maria de Almeida Carvalho, Lívio Matheus Oliveira Silva, Ney Cristian Amaral Boa-Sorte, Mônica Viegas Andrade   +7 more
doaj   +1 more source

Reduction in Perioperative Risk in Patients with Spinal Muscular Atrophy Following the Release of Disease-Modifying Therapies: An Analysis of the National Surgical Quality Improvement Program Database

Children
Background/Objectives: Spinal muscular atrophy (SMA) is a progressive neurodegenerative disease resulting in proximal muscle weakness and paralysis. SMA treatment has radically changed in the past 10 years thanks to the development of novel therapies ...
Erin Toaz, Nisha Pinto, Keith Kilner, Eric Cheon   +3 more
doaj   +1 more source