Publications by authors named "Imelda Jm de Groot"
Article Synopsis
- Using real-world data (RWD) and natural history data (NHD) for evaluating drugs in Duchenne muscular dystrophy (DMD) offers a solution to the difficulties of enrolling participants in placebo-controlled trials.
- A study compared the changes in the North Star Ambulatory Assessment (NSAA) scores between placebo trial arms and RWD/NHD data, revealing that the differences were minor before and after adjusting for baseline factors.
- The findings suggest that RWD/NHD external controls are appropriate for DMD drug evaluations, as they align with earlier results and provide a solid method for accounting for prognostic factors.
Editorial: Motor Function Measure in Duchenne Muscular Dystrophy.
Eur J Paediatr Neurol
January 2022
Background: "Use it or lose it" is a well known saying which is applicable to boys with Duchenne Muscular Dystrophy (DMD). Besides the direct effects of the muscular dystrophy, the increasing effort to perform activities, the fear of falling and the use of personal aids indirectly impair leg and arm functions as a result of disuse. Physical training could oppose this secondary physical deterioration.
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