Celebrating the Pompe Community
In recognition of the third annual International Pompe Day, and the 10 Year Anniversary of the approval of Myozyme, Genzyme developed a video highlighting how far the Pompe community has come in the last 10 years, and our hopes for the future. Please find the video...
Press Release: International Pompe Day 2016
On the third annual International Pompe Day, the Pompe Community turned its attention to the importance of Early Diagnosis. For Immediate Release April 15, 2016 will be the Third Annual International Pompe Day. The International Pompe Association (IPA), with the...
Pompe Registry – A Decade of Making a Difference
In 2014, the Pompe Registry recognized and celebrated an important milestone: the 10-year anniversary of the Registry. Started in 2004 and sponsored by Genzyme, a Sanofi company, the Pompe Registry was established as a global, voluntary, observational database to...
Sanofi Genzyme Presents Results from Phase 1/2 Study of Investigational Second-Generation Therapy
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...
Inspired By Pompe Cookbooks available for Sale!
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...
BioMarin Announces Interim Analysis of INSPIRE Clinical Trial in Pompe Disease
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...
2016 PCMA of Texas Pull for Pompe!
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...
Amicus Therapeutics Commences Phase 1/2 Study of Novel ERT for Treatment of Pompe Disease
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...
2015 Helen Walker Research Grant Awarded
The sixth annual AMDA Research Grant (the 2015 Helen Walker Research Grant) was awarded to Dr. Rosa Puertollano and Dr. Nina Raben for their Project: “Curcumin: Can it promote cellular clearance in Pompe disease?.” The aim of this project is to test the possibility of...
2015 AMDA International Pompe Patient and Scientific Conference-Update
For the twentieth (20th) anniversary of the AMDA, we will be hosting an International Patient and Scientific Conference in San Antonio, Texas from October 30, 2015 - November 1, 2015. The Conference will begin on Friday evening (October 30th) with a Welcome Dinner,...
2015 Helen Walker Research Grant for Pompe Disease Now Accepting Applications
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 fundraiser hosted by the Precast Manufacturer’s Association of Texas (PCMA of Texas) on April 18,...
Registration now open for 2015 AMDA International Pompe Patient and Scientific Conference
We are excited to announce that Registration is now open for the 2015 AMDA International Pompe Patient and Scientific Conference! The Conference will be held in San Antonio, Texas from October 30-November 1, 2015. In order to assist with Conference planning, we ask...
Genzyme’s Pompe Community Program Update for International Pompe Day
Please click here to read the press release.
Pompe Program to Air on Lifetime’s Access Health on April 15, 2015
The AMDA is excited to announce that in collaboration with Genzyme Corporation, AMDA President and IPA Chair, Tiffany House, participated in a segment on Pompe Disease awareness for Access Health on Lifetime Television! This program will air on April 15th, 2015 in...
Pompe Disease to be Added to Recommended Newborn Screening Panel in the United States
The AMDA is very excited to announce that the U.S. Pompe Community is one step closer to having newborn screening become a reality for Pompe disease! On March 2, 2015, the Secretary of Health and Human Services, Sylvia Burwell, accepted the recommendation to add Pompe...
Date Set for 5th Annual PCMA of Texas’ Pull for Pompe!
The PCMA of Texas invites you to enjoy a day of family fun benefiting Acid Maltase Deficiency, also known as Pompe Disease. This event is designed for all ages and ability levels. It will be held at the National Shooting Complex in San Antonio, Texas. Please click...
Australian Pompe’s Association Welcomes Approval of Treatment for Young Australians with Pompe
Press Release by Australian Pompe Association (APA) 17th February 2015 The Australian Pompe’s Association (APA) welcomes the news that the Minister for Health Sussan Ley has approved treatment for young Australians with Pompe disease. As of the 1st February 2015, the...
Second Annual International Pompe Day!
April 15th, 2015 will mark the Second Annual International Pompe Day! Last year the Pompe Community worked together to raise awareness of Pompe Disease, and showed the world how Strong our Community can be. Please click here for a summary of the events and activities...
Update on Biomarin’s Pompe Clinical Program—January 2015
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
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...