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

• Main Authors: Craig M McDonald (Author), Jessica R Marden (Author), Perry B Shieh (Author), Brenda L Wong (Author), Henry Lane (Author), Adina Zhang (Author), Ha Nguyen (Author), Molly Frean (Author), Panayiota Trifillis (Author), Karyn Koladicz (Author), James Signorovitch (Author)
• Format: Book
• Published: Becaris Publishing Limited, 2023-02-01T00:00:00Z.
• Subjects: article
• Online Access: 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...