Outcome measures in Duchenne muscular dystrophy

Author(s):
Naarding, K.J.
Published
2023
Publication type
Doctoral Thesis

Abstract

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 dystrophyOutcome measuresAmbulantNon-ambulantClinically relevant
Defense date
2023-03-16
Language
English
Supervisor(s)
Verschuuren, J.J.G.M.
Advisor(s)
Niks, E.H.; Kan, H.E.
Committee member(s)
Dijk, J.G van; Geijsen, N.; Groot, I.J.M. de; Strijkers, G.J.
Qualification
Ph.D.
Degree granting institution
Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University, Leiden
hdl
1887/3571791
ISBN(s)
9789464692075