Outcome measures in Duchenne muscular dystrophy

Naarding, K.J. (2023)

Doctoral Thesis

The overall aim of this thesis was to identify outcome measures in Duchenne muscular dystrophy (DMD), specifically for non-ambulant patients, that are able to detect a clinically relevant difference in a relatively short period of time compatible with the duration of a clinical trial. The use of such outcome measures could lead to smaller sample sizes in such trials with a lower burden for patients.

• Duchenne muscular dystrophy
• Outcome measures
• Ambulant
• Non-ambulant
• Clinically relevant

• All authors: Naarding, K.J.
• Supervisor: Verschuuren, J.J.G.M.
• Co-supervisor: Niks, E.H.; Kan, H.E.
• Committee: Dijk, J.G van; Geijsen, N.; Groot, I.J.M. de; Strijkers, G.J.
• Qualification: Doctor (dr.)
• Awarding Institution: Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University
• Date: 2023-03-16
• ISBN (print): 9789464692075