A uncommon inherited neurodegenerative disease of childhood and adolescence.

These data could possibly be provided in order to support a resubmission in this indication in the near future. More info about Zavesca and the state of the scientific assessment at the time of withdrawal will be made available in a question-and-answer record. This document, together with the withdrawal letter from the business, will be published on the EMEA site in due course. For further information, please visit:.. Actelion withdraws its application for an expansion of indication for Zavesca The European Medications Agency has been formally notified by Actelion of its decision to withdraw its application for an extension of indication for the centrally authorised medication Zavesca .In Duchenne muscular dystrophy , the mutated dystrophin protein does not anchor to its membrane glycoprotein complex correctly. And without this anchoring, muscle cells experience serious contraction-induced harm. Sarcospan is part of the anchoring complex, but because mice without sarcospan don’t seem any worse because of its absence, it hasn’t received much interest. Sarcospan’s structure, however, suggests it could help stabilize the membrane complicated, so the authors decided to test the effects of increasing sarcospan expression in a DMD mouse model. Utrophin is restricted to the neuromuscular junction normally, where it serves a job similar to that of dystrophin. The extra sarcospan prompted higher degrees of utrophin in the cell, but not by increasing its expression.