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...

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...

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...

The 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. It will be held at the National Shooting Complex in San Antonio, Texas. Please click...

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: “Mitochondria and Autophagy as Therapeutic Targets for Acid Maltase Deficiency.” Lysosomes are responsible for...

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)...

AT2220 Co-Administered with Enzyme 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...

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...

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...

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...