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Update on Biomarin’s Pompe Clinical Program—January 2015

Jan 6, 2015 | NEWS

BioMarin are pausing further recruitment for their BMN-701 clinical trial and have provided this programme update: January 2015 BioMarin is pleased to update the Pompe patient community on the progress made in our programme to develop a treatment for people living...

2014 Helen Walker Research Grant Awarded

Dec 15, 2014 | NEWS

The fifth annual AMDA Research Grant (the 2014 Helen Walker Research Grant) was awarded to Dr. Giancarlo Parenti with the Federico II University in Naples, Italy for his Project: “Analysis of Circulating and Tissue Specific microRNAs in Pompe Disease.” Despite...
Design And Performance Of Clinical Trials: An Overview

Design And Performance Of Clinical Trials: An Overview

Oct 20, 2014 | PAST WEBINARS

Webinar Details Title: Design and Performance of Clinical Trials: An Overview Date: Monday, October 20, 2014 Speaker:Anne Zajicek, MD, PharmD, FAAP, Chief, Obstetric and Pediatric, Pharmacology and Therapeutics Branch, National Institute of Child Health and Human...

United States Pompe Community Update from Genzyme

Oct 17, 2014 | NEWS

Genzyme would like to share with you an important update regarding the recent United States (U.S.) Food and Drug Administration’s (FDA) approval of an expanded label for Lumizyme® (alglucosidase alfa) manufactured at the 4000L scale and its impact on Myozyme®...