Clinical decision making around commercial use of gene and genetic therapies for spinal muscular atrophy.
Neurotherapeutics
; 21(4): e00437, 2024 Jul.
Article
em En
| MEDLINE
| ID: mdl-39241317
ABSTRACT
Spinal muscular atrophy is no longer a leading cause of inherited infant death in the United States. Since 2016, three genetic therapies have been approved for the treatment of spinal muscular atrophy. Each therapy has been well studied with robust data for both safety and efficacy. However, there are no head-to-head comparator studies to inform clinical decision making. Thus, treatment selection, timing, and combination therapy is largely up to clinician preference and insurance policies. As the natural history of spinal muscular atrophy continues to change, more data is needed to assist in evidence-based and cost-effective clinical decision making.
Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Atrofia Muscular Espinal
/
Terapia Genética
/
Tomada de Decisão Clínica
Limite:
Humans
Idioma:
En
Ano de publicação:
2024
Tipo de documento:
Article