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 …

Natural history (NH) controls can be attractive in rare diseases such as DMD because they
enable smaller drug trials with faster enrollment. Wellknown challenges include potential …

Since new therapeutic interventions are pounding on the front door for clinical application in
Duchenne muscular dystrophy (DMD), a quick and determined approach is needed to …

Abstract In June 2010, 25 representatives from Europe and the US met in Washington, DC,
USA, to discuss clinical outcome measures in Duchenne muscular dystrophy (DMD) in the …

Event-driven clinical trials, commonly used in oncology, offer several benefits over
continuous outcome measures: i) each patient is assessed individually, ii) after the event …

Using external controls based on real-world or natural history data (RWD/NHD) for drug
evaluations in Duchenne muscular dystrophy (DMD) is appealing given the challenges of …

There are multiple avenues for therapeutic development in Duchenne muscular dystrophy
(DMD), which are highlighted in the first section of this report for the “10 years of Clinical …

Background: International care guidelines for Duchenne muscular dystrophy (DMD) were
published in 2010, but compliance in clinical practice is unknown. Objective: The objective …

Duchenne muscular dystrophy (DMD) is a lethal inherited neuromuscular disorder caused
by mutations in the dystrophin gene and subsequent absence of the dystrophin protein, for …

A multi-domain progression endpoint has the potential to broaden DMD clinical trial
enrollment and support integrated assessments of treatment efficacy representing different …