Results 111 to 120 of about 2,700 (197)

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

Type I spinal muscular atrophy and disease modifying treatments: a nationwide study in children born since 2016 [PDF]

Background The advent of disease-modifying treatments (DMT) has changed natural history in 5q Spinal muscular atrophy (SMA). The aim of this study was to report survival and functional aspects in all the Italian type I children born since 2016.
Cavallina, Ilaria, Leone, Daniela, Mastella, Chiara, Ricci, Federica   +3 more
core   +4 more sources

A new score combining compound muscle action potential (CMAP) amplitudes and motor score is predictive of motor outcome after AVXS-101 (Onasemnogene Abeparvovec) SMA therapy

, 2023
Rémi Barrois, Christine Barnérias, E. Deladrière, V. Leloup-Germa, Brian Tervil, Frédérique Audic, C. Boulay, Claude Cancés, Pascal Cintas, Jean‐Baptiste Davion, Caroline Espil‐Taris, V. Manel, Yann Péréon, Julie Piarroux, S Roy, C. Vuillerot, Ulrike Walther‐Louvier, Isabelle Desguerre, Cyril Gitiaux   +18 more
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

Quality of Life of Children With Spinal Muscular Atrophy: Parents’ Perspectives in Light of New Treatments [PDF]

, 2020
Purpose: To directly compare parents’ perspectives of the quality of life of their children with Spinal Muscular Atrophy (SMA) who received supportive care, nusinersen (Spinraza®), onasemnogene abeparvovec-xioi (Zolgensma®), or both nusinersen and ...
Tallas, Analyssa R.
core   +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

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

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