Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status

• Autores principales: Craig M McDonald (Autor), Jessica R Marden (Autor), Perry B Shieh (Autor), Brenda L Wong (Autor), Henry Lane (Autor), Adina Zhang (Autor), Ha Nguyen (Autor), Molly Frean (Autor), Panayiota Trifillis (Autor), Karyn Koladicz (Autor), James Signorovitch (Autor)
• Formato: Libro
• Publicado: Becaris Publishing Limited, 2023-02-01T00:00:00Z.
• Materias: article
• Acceso en línea: Connect to this object online.

Aim: To examine benefits of corticosteroids for Duchenne muscular dystrophy (DMD) by age and disease progression. Methods: Data from daily steroid users (placebo-treated) were pooled from four phase 2b/3 trials in DMD. Outcomes assessed overall and among subgroups included changes from baseline to 4...