May 13, 2012—Program Update from BioMarin. For more information about the BioMarin trials please contact Sean McCarthy of BioMarin at: smccarthy@bmrn.com. The AMDA is also available to help answer any questions or concerns.  Please contact Tiffany House or Marsha...

Order your 2011 AMDA/IPA Conference DVD Today! This set of DVDs includes all of the presentations and Question and Answer Sessions on October 8-9th, 2011. To order your DVDs, please send an email with your mailing address and the number of copies you would like to...

March 19, 2012 Community Update for Pompe Families in the United States who Utilize Myozyme® (alglucosidase alfa) Produced at the 160 L Manufacturing Scale Download complete article

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. The 2012 Pull for Pompe will take place on Saturday, April 21, 2012 at the National...

As some of you are probably aware, Genzyme has announced that, due to production and supply constraints, starting March 30, 2012 the 160 liter Myozyme (which is ONLY used in the United States) will no longer be shipped to patients over the age of 12 months. This ONLY...

Excerpt from August 30, 2010 press release . . . BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for BMN-701, a novel fusion of insulin-like growth factor 2 and...

Hi All, The AMDA has been working with Pfizer on supporting some proposed legislation in California that is intended to streamline the process to get prior authorization of medication.  The proposed Bill is SB 866. This Bill could be taken up by the House and Senate...

Genzyme would like to provide an update to the US Pompe community regarding the transition to commercial LumizymeTM (alglucosidase alfa) from the ATAP (Alglucosidase Alfa Temporary Access Program) in the United States. As a reminder, Lumizyme was approved by the US...

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 aimed at promoting research into Pompe Disease. If you are interested in this opportunity,...

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 private fundraiser aimed at promoting research into Pompe Disease. If you are interested in this...