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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More and effects of enzymeA protein that helps the body carry out chemical reactions. More 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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More and the effects of enzymeA protein that helps the body carry out chemical reactions. More replacement therapy. During this webinar we will discuss what we have learned about Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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 DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.” Respiratory insufficiencyA condition in which the breathing muscles are too weak to support normal breathing needs. More 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 DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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 DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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...
2013 PCMA of Texas’s Pull for Pompe set for April 20, 2013!
The PCMA of Texas invites you to enjoy a day of family fun benefitting Acid Maltase Deficiency, also known as Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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...
2012 AMDA Research Grant Awarded
The AMDA is excited to announce that the 2012 AMDA Research Grant Recipient is Dr. Nuno Raimundo of the University of Goettingen in Germany for his Project: “MitochondriaParts of the cell that produce energy. More and AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. More as Therapeutic Targets for Acid Maltase Deficiency.” LysosomesA small part of the cell that acts like a recycling center, helping break down waste materials. More are responsible for...
Amicus Announces Positive Results from Phase 2 Chaperone-ERT Co-Administration Study
CRANBURY, NJ, January 4, 2013 – Amicus Therapeutics today announced positive preliminary results from all 4 dose cohorts in a Phase 2 study (Study 010) to evaluate the safety and pharmacokinetic (PK) effects of the pharmacological chaperone AT2220 (duvoglustat HCl)...
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease
AT2220 Co-Administered with EnzymeA protein that helps the body carry out chemical reactions. More Replacement Therapy Second Phase 2 Pharmacological Chaperone-ERT Co-Administration Study CRANBURY, NJ, US, December 1, 2011 – Amicus Therapeutics, a biopharmaceutical company at the forefront of developing therapies for rare...
Pompe Community Loses Valued Advocate
On the 30th November 2012, Helen Walker OAM (Member of the Order of Australia) passed away. Helen was a board member of the International Pompe Association (IPA) and President of the Australian Pompe’s Association. Helen passed away at the age of 72 after having...
News 2012 AMDA/Genzyme Patient Diagnosis Survey Results
The path to diagnosis can be long and complicated and patients are frequently misdiagnosed along the way. In an effort to improve diagnostic accuracy, the AMDA and Genzyme collaborated on a questionnaire project in the first-quarter of 2012. The goal of the...
News Dr. Arnold Reuser Decorated by Queen of Netherlands
On Thursday, August 30, 2012 Dr. Arnold Reuser was decorated as a Knight in the Order of the Netherlands Lion by decree of Queen Beatrix of the Netherlands. The decoration was presented at the conclusion of the “Passion for Pompe” Program at the Arminius Church in...