Results 121 to 130 of about 3,107 (205)

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

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  

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

FRAME: Framework for Real‐World Evidence Assessment to Mitigate Evidence uncertainties for efficacy/effectiveness – an evaluation of regulatory and health technology assessment decision making [PDF]

Real‐World Evidence (RWE) is increasingly used in submissions to regulatory agencies and health technology assessment bodies (HTAbs) to support the efficacy and effectiveness of new medicines and indications.
Bolot, Paul, Candore, Gianmario, Chavez‐Montoya, Danitza, Civitelli, Diego, Kanavos, Panos G., Lloyd, Anna, Martin, Claire, Mills, Mack J., Sculpher, Mark, Soriano Gabarró, Montse, Suter, Annabel, Wasem, Juergen, Wolf, Birgit   +12 more
core   +2 more sources

Onasemnogene Abeparvovec (AVXS-101) for the Treatment of Spinal Muscular Atrophy

, 2021
Aimen Naveed, Hillary Calderon
openalex   +2 more sources

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

Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy [PDF]

, 2021
Jerry R. Mendell, Samiah Al-Zaidy, Kelly J. Lehman, Markus McColly, Linda Lowes, Lindsay N. Alfano, Natalie F. Reash, M. Iammarino, Kathleen R. Church, A. de Kleyn, Matthew N. Meriggioli, Richard Shell   +11 more
openalex   +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

Review of cardiac safety in onasemnogene abeparvovec gene therapy: translation from preclinical to clinical findings [PDF]

, 2022
Deepa H. Chand, Rui Sun, Karim Diab, Damien Kenny, Francis Funyuy Tukov   +4 more
openalex   +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