June 1st, 2008 This E-mail was from Debra Miller Dear Friends, For those of you who have been supporters of CureDuchenne, you will appreciate the excitement and hope we have. Four years ago, we partnered with Prosensa to make exon skipping a reality. It was through the generous encouragement and donations of many of you, that we are able to make this announcement today.
Please remember that this particular drug will only help a small percentage of Duchenne boys. We must keep up the intensity to save every one of our boys. Now, more than ever, we need your support and your prayers so that today’s Duchenne boys can have a tomorrow. Thank you from the bottom of this mom ‘s heart, Debra PRESS RELEASE, May 5th, 2008 Prosensa announces the start of an international multi-center phase I/II clinical study with ‘smart drug’ PRO051 in patients with Duchenne Muscular Dystrophy On May 5th, 2008, Prosensa announces that it has started a phase I/II study to explore the effect, safety and tolerability of systemic injections of PRO051 in Duchenne Muscular Dystrophy (DMD) patients. This trial is performed in collaboration with UZ Leuven (Belgium), the Queen Silvia Children’s hospital (Sweden) and the LUMC (the Netherlands). The UZ Leuven and the Queen Silvia Children’s hospital have already started to enroll patients. In this study, an important parameter will be the presence of dystrophin in muscle biopsies, the protein missing in patients with Duchenne Muscular Dystrophy (DMD). This clinical trial in patients with DMD uses an antisenseolig oribonucleotide, a ‘smart drug’ removing an unwanted segment of the faulty DMD gene product, and represents a novel approach to combat genetic diseases like DMD. |