The FDA and Gene Therapy for Duchenne Muscular Dystrophy.
JAMA
; 331(20): 1705-1706, 2024 05 28.
Article
en En
| MEDLINE
| ID: mdl-38691382
This Viewpoint examines the appropriateness of FDA accelerated approval of novel gene therapies to treat boys with Duchenne muscular dystrophy following clinical trials with surrogate outcomes that did not demonstrate net benefits.
Texto completo:
1
Bases de datos:
MEDLINE
Asunto principal:
United States Food and Drug Administration
/
Terapia Genética
/
Distrofia Muscular de Duchenne
Límite:
Child
/
Child, preschool
/
Humans
/
Male
País/Región como asunto:
America do norte
Idioma:
En
Revista:
JAMA
Año:
2024
Tipo del documento:
Article