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
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. Os pacientes com AME 5q podem ter uma ou mais cópias do gene SMN2, homólogo...
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| Main Authors: | 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 |
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| Format: | Article |
| Language: | English |
| Published: |
Instituto Nacional de Assistência Farmacêutica e Farmacoeconomia
2024-11-01
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| Series: | Jornal de Assistência Farmacêutica e Farmacoeconomia |
| Subjects: | |
| Online Access: | https://ojs.jaff.org.br/ojs/index.php/jaff/article/view/972 |
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