Mar 3, 2016 | NEWS
Results support beginning pivotal Phase 3 clinical trial in Q2 2016 PARIS—(BUSINESS WIRE)—Sanofi and its specialty care global business unit Sanofi Genzyme today presented data from NEO1, its Phase 1/2 clinical study evaluating the investigational novel enzyme...
Feb 5, 2016 | NEWS
The AMDA is excited to announce that “Inspired by Pompe—High Protein, Reduced Carb Cooking” Cookbooks are availble for sale. This cookbook was written by fellow Pompe patient Sharon Krueger, and all proceeds will go towards the AMDA. Click here to view the Preface and...
Jan 11, 2016 | NEWS
Reveglucosidase Alfa Trends Positive in Respiratory Muscle Strength and Endurance Endpoints in Patients Previously Treated With Enzyme Replacement Therapy SAN RAFAEL, Calif., Jan. 11, 2016 (GLOBE NEWSWIRE)—BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) today announced...
Jan 7, 2016 | NEWS
The AMDA is excited to announce that the 2016 PCMA of Texas Pull for Pompe will take place on Saturday, April 16 at the National Shooting Complex in San Antonio, Texas! This is the 6th Annual Pull for Pompe hosted by the PCMA of Texas. Since it’s launch in 2011, over...
Dec 22, 2015 | NEWS
CRANBURY, N.J., Dec. 22, 2015 (GLOBE NEWSWIRE)—Amicus Therapeutics (Nasdaq:FOLD), a biopharmaceutical company at the forefront of rare and orphan diseases, today announced that its investigational new drug (IND) application, submitted to U.S. Food and Drug...