AMDA 2010 Research Grant Opportunity
The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This grant opportunity was made possible by a private fundraiser aimed at promoting research into Pompe Disease. If you are interested in this...
Upcoming Event: Respiratory Muscle Strength Training in Pompe Disease Webinar
On June 6, 2011 the AMDA will host a webinar on Respiratory Muscle Strength Training in Pompe disease at 12:00pm (ET); 11:00am (CT); 10:00am (MT); 9:00am (PT). The Guest Speaker is: Harrison N. Jones, PhD, BRS-S, CCC-SLP, Assistant Professor, Department of Surgery,...
PCMA of Texas hosting “Pull for Pompe” Fundraiser
PCMA of Texas invites you to enjoy a day of family fun benefitting Acid Maltase Deficiency, also known as Pompe Disease. This event is designed for all ages and ability levels. Proceeds from the event will go to the Acid Maltase Deficiency Association (AMDA). For...
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...
BMN-701:Potentially More Effective Treatment Option for Pompe
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Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
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Autophagy and It’s Role In Pompe Disease
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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...
AMDA nominates Drs. Reuser and van der Ploeg to Rare Disease Day Hall of Fame
By: Tiffany House--AMDA President posted on: March 01, 2010 The AMDA has nominated Dr. Arnold Reuser and Dr. Ans van der Ploeg to the Rare Disease Day Research Hall of Fame. Drs. Reuser and van der Ploeg have spent decades researching Pompe disease, and from their...
Editorial: Extraordinary Measures or an Extraordinary Tale? (Update)
By: Tiffany House--AMDA President posted on: February 15, 2010 What happened to the TRUTH? In the wake of the recent article by Quest Magazine (January 2010) and the upcoming film “Extraordinary Measures” I feel that I must say something as to the accuracy of 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...
ATAP Inclusion/Exclusion and List of Study Sites
This information was complied from the information provided on clinicaltrials.gov. To read, please download file by clicking here.
December 2009 Pompe Program Update
Genzyme would like to provide an update to the US Pompe community about the status of the Alglucosidase Alfa Temporary Access Program (ATAP) and the application for Lumizyme™ (alglucosidase alfa) approval in the United States. In a press release dated December 3,...