Date: 2014-09-24
Type of information: Publication of results in a medical journal
phase:
Announcement: publication of results in Plos One
Company: Prosensa (The Netherlands)
Product: semi-automated image analysis method
Action mechanism:
Disease: Duchenne muscular dystrophy (DMD) Becker\'s muscular dystrophy (BMD)
Therapeutic area: Rare diseases - Neuromuscular diseases
Country:
Trial details:
Latest
news: * On September 24, 2014, Prosensa announced that the results of its research into developing an accurate and reproducible method for the measurement of dystrophin in patients with Duchenne muscular dystrophy (DMD) and Becker\'s muscular dystrophy (BMD) have been published in the online peer reviewed journal PLOS ONE (http://bit.ly/ZJ8ShM). The publication, by Chantal Beekman et al from Prosensa, describes the Company\'s semi-automated image analysis method, which was shown to be objective (operator independent), reproducible (in multiple samples and experiments) and sensitive for assessing dystrophin levels by immunofluorescence in muscle biopsies from BMD and DMD patients in natural history studies or clinical studies with compounds aiming to restore dystrophin expression. This method has also been tested as part of an international consortium initiative, the Biochemical Outcome Measures study group, which focuses on the optimization of dystrophin assays. As dystrophin expression is variable between muscle groups and even within the same muscle, demonstration of a pharmacodynamic effect requires comparison of two biopsies pre- and post-treatment, taken from the same muscle, and sampled from areas of similar disease state. Furthermore, determining intensity of individual fibers and its distribution across the biopsy yields a more informative and objective measure of dystrophin expression than counting fibers.
