TOP NEWS
2023 Helen Walker Grant Recipient
Project title: Autophagy control of glycogen in skeletal muscle This year, the AMDA was fortunate to receive 13 grant applications focusing on both clinical research and basic science. It has been a challenge to select one. Dr. Wyant was chosen because he has embarked...
OTHER NEWS
Cincinnati Children’s Hospital Study on Speech, Language and Swallowing Problems
Cincinnati Children’s Hospital is conducting a study about the speech, language and swallowing problems in infants and children with Pompe disease. The study involves a survey which can be completed online that asks a variety of questions about the speech, language...
AMDA Webinar on Genzyme’s Next Generation Pompe Treatment: Clinical Development Overview
The AMDA is hosting yet another webinar for the Pompe community on Feb 3, 2011. Title: Genzyme’s Next Generation Pompe Treatment: Clinical Development Overview Date: Thursday, February 3, 2011 Time: 8:00 pm (ET); 7pm (CT); 6pm (MT); 5pm (PT) Guest Speakers: Edward...
Genzyme to Build Additional Plant to Support Growth of Myozyme® and Lumizyme®
CAMBRIDGE, Mass.—Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term growth of Myozyme® and Lumizyme® for Pompe disease. The company held a ceremony today in Geel to mark the start of...
BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease
Jan. 19, 2011—BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe...
2010 AMDA Research Grant Awarded to Dr. Andrea Amalfitano
In 2010 the AMDA solicited grant applications from researchers around the world. After careful consideration and consultation with our Scientific Advisory Board, Dr. Amalfitano of Michigan State University was selected as the recipient of the First Annual AMDA...
Pompe Patient Meeting Hosted by Columbia University
Dr. Maryam Banikazemi of Columbia University is hosting a meeting for Pompe patients on July 16, 2010 in New York, New York. The expert panel of speakers will include Drs. J. Bach, A. Slonim, T. Goldberg, and M. Banikazemi. For more details, please download meeting...
Pompe Program Update—FDA Approves Lumizyme
On May 24, 2010 the FDA approved Genzyme’s Lumizyme for the treatment of patients with Pompe disease who are over 8 years of age and do not have evidence of cardiac hypertrophy. For more information on the approval and how to access treatment, please read the Pompe...
The FDA Approves Lumizyme for Late-Onset Pompe Disease
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has granted U.S. marketing approval for LumizymeTM (alglucosidase alfa), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the...
Pompe Disease: The Real Story
To read a factual account of the true history of Pompe Disease, please visit Pompe Disease—The Real Story.
AMDA Press Release Regarding Extraordinary Measures
The AMDA issued a press release regarding the film, Extraordinary Measures. The press release can be found here. In addition, the AMDA created a one-page fact sheet regarding Pompe disease to help raise awareness of the condition. That fact sheet can be found...
Acid Maltase Deficiency Association
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