Article

Core protocol development for phase 2/3 clinical trials in the leukodystrophy Vanishing White Matter

2023-02-07

Abstract excerpt

<h4>Background: </h4> The leukodystrophy “Vanishing White Matter” (VWM) is an orphan disease with neurological decline and high mortality. Currently, VWM has no approved treatments, but advances in understanding pathophysiology have led to identification of promising therapies. Several investigational medicinal products are either in or about to enter clinical trial phase. Clinical trials in VWM pose serious chall...

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Literature Corpus work: dda2fbd5-ebbe-5258-967a-87c6632b593c
DOI: 10.21203/rs.3.rs-2446334/v1

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