Results 81 to 90 of about 2,067 (154)

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

CASE REPORT: Liver Failure in a 4-month-old male with SMA type 2 after gene therapy/Onasemnogene abeparvovec (Zolgensma) [PDF]

, 2023
Introduction: Spinal muscular atrophy (SMA) is characterized by muscle weakness and atrophy resulting from progressive degeneration of the anterior horn cells in the spinal cord and the brain stem nuclei. The onset of weakness ranges from before birth to
Hager, Juliana, Paul, Dustin J.
core  

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

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

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

Vaccination proposal for patients on onasemnogene abeparvovec therapy

European Journal of Paediatric Neurology
The approval of disease-modifying treatment in spinal muscular atrophy made the condition less severe. The course of the disease changed, but some new concerns occurred with the different new therapies. The side effects of onasemnogene aboparvovec therapy can raise differential diagnostic challenges and necessitate immune therapy, leading to ...
Sarolta Dobner, Andrea Kulcsár, Zoltán Liptai, Zsuzsanna Vojnisek, Tamás Constantin, Léna Szabó   +5 more
openaire   +2 more sources