Jul 22, 2013 | NEWS
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,...
Jul 16, 2013 | NEWS
To read press release, please click here.
Jul 1, 2013 | NEWS
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...
May 17, 2013 | NEWS
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...
May 13, 2013 | NEWS
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...
Mar 22, 2013 | NEWS
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...
Feb 16, 2013 | NEWS
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...
Jan 5, 2013 | NEWS
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...
Jan 4, 2013 | NEWS
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)...
Dec 1, 2012 | NEWS
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...
