Kate Bushby
Neurologin,
Genetikerin
Werke:
- Ataluren treatment of patients with nonsense mutation dystrophinopathy: ataluren for dystrophinopathy
- How reference networks develop, implement, and monitor guidelines
- A checklist for clinical trials in rare disease: obstacles and anticipatory actions—lessons learned from the FOR-DMD trial
- The TREAT-NMD care and trial site registry: an online registry to facilitate clinical research for neuromuscular diseases