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 DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart., 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 microRNAsSmall molecules that help control how genes are expressed in cells. in Pompe Disease.” Despite...
Design And Performance Of Clinical Trials: An Overview
Title: Design and Performance of Clinical TrialsA research study that tests new treatments or approaches in people.: 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 Development (NICHD)
United States Pompe Community Update from Genzyme
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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.) manufactured at the 4000L scale and its impact on Myozyme®...
FDA and Genzyme Announce Label Expansion of Lumizyme in United States
To the United States Pompe Community: On August 1, 2014 the US Food and Drug Administration (FDA) and Genzyme issued press releases regarding the expansion of the Lumizyme® label (the enzymeA protein that helps the body carry out chemical reactions. replacement therapy for Pompe disease produced at the 4000L manufacturing...
2014 Helen Walker Research Grant 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 5,...
BioMarin Doses First Patient in Phase 3 INSPIRE Trial with BMN 701 for the Treatment of Pompe
San Rafael, Calif., May 27, 2014 – BioMarin Pharmaceutical Inc. announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe disease. BMN 701 is a novel fusion protein of insulin-like...
AMDA to Celebrate First International Pompe Day!!
Update: May 4, 2014 THE AMDA TO CELEBRATE THE FIRST INTERNATIONAL POMPE DAY WITH COMMEMORATIVE BOOK April 15, 2014 marked the International Pompe Association’s (IPA) first annual International Pompe Day. In honor of the First International Pompe Day, the Acid Maltase...
The Contribution Of The IPA Survey To Our Understanding Of The Natural Course Of Pompe Disease
Title: The contribution of the IPA survey to our understanding of the natural course of Pompe disease and effects of enzyme therapy: Ten years of the IPA survey
Date: Thursday, February 27, 2014
Speaker:
J.C. van der Meijden, Center for Lysosomal and Metabolic diseases, Erasmus MC University Medical Center, Rotterdam, the Netherlands
Webinar Overview:
For more than ten years Pompe patients, from all over the world, participate in the International Pompe Association / Erasmus MC Pompe survey on a yearly basis. During this period we have gathered a vast amount of information on the natural course of Pompe disease and the effects of enzyme replacement therapy. During this webinar we will discuss what we have learned about Pompe disease by working with the Survey. Furthermore, we will discuss the importance of the survey and goals for the future. At the last part of the webinar there is an opportunity for participants to ask questions to the researchers.
2014 AMDA Pull for Pompe
On Saturday April 5, 2014 the AMDA will host the Fourth Annual Pull for Pompe at the National Shooting Complex in San Antonio, Texas. Over the last three years over $380,000.00 (USD) has been raised through this event and all proceeds go towards the AMDA Research...
Update on Genzyme’s neo-GAA Pompe Clinical Development Program
To read the Program Update, please click here.
2013 Helen Walker Research Grant Awarded
The fourth annual AMDA Research Grant (named the 2013 Helen Walker Research Grant) was awarded to Dr. Barbara Smith with the University of Florida for her Project: “[Diaphragmatic] Pacing in Subjects with Pompe Disease.” Respiratory insufficiencyA condition in which the breathing muscles are too weak to support normal breathing needs. is a serious, and...
2013 Helen Walker Research Grant 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 20,...
In Memoriam: Helen Walker
By: Tiffany House posted on: July 22, 2013 On November 30, 2012 the Pompe Community lost one of its most valued Advocates: Helen Walker. Helen was the President of the Australian Pompe Association (APA) and a Board Member of the International Pompe Association. She...
Update on Genzyme’s neo-GAA Program
To read press release, please click here.
2013 PCMA of Texas’s Pull for Pompe a HUGE Success!
On April 20, 2013 the Precast Concrete Manufacturers’ Association of Texas (PCMA of Texas) hosted its Third Annual “Pull for Pompe” fundraiser. Two hundred (200) shooters and one hundred spectators (100) enjoyed a day of friendly competition and family fun at this...
Federal Advisory Committee Recommends Pompe Disease for Newborn Screening
WASHINGTON – May 17 2013 – In a long awaited meeting, the Secretary’s Discretionary Advisory Committee for Heritable Disorders in Newborns and Children (DACHDNC), in a vote of 11 – 2, recommended the addition of Pompe Disease to the recommended uniform newborn...
Callidus Biopharma Announces $ 4 .6 million in Series A Financing to Pursue Orphan Disease Therapies
DOYLESTOWN, PA (May 13, 2013)–Callidus Biopharma, Inc., a development-stage biotechnologycompany focused on creating breakthrough biologic drugs for a range of orphan diseases, announced today that it has closed on $4.6 million in Series A financing led by two...
BioMarin to Advance BMN-701 for Pompe Disease to Next Phase of Development
San Rafael, Calif, March 19, 2013 – BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today results from POM-001, the Phase 1/2 trial for BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha-glucosidase (IGF2-GAA) for the treatment of...