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...
By: –A Late-onset Pompe patient posted on: May 14, 2012 Something to think about…… A cause that is very near and dear to my heart is organ donation. I have a close relative that under went a kidney transplant a few years ago that saved his life. I have the...
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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.) Produced at the 160 L Manufacturing...
Webinar Details Title: Needle Core Muscle Biopsy Date: Wednesday, March 7, 2012 Speaker:Mark Tarnopolsky, MD, PhD, FRCP (C) Webinar Overview:Traditionally most Neuromuscular Clinics have used an open muscle biopsy for pathological diagnosis. Many Neuromuscular...
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...
2020AOn October 7-9, 2011, 150 Pompe patients and their families, scientists, doctors, and industry representatives from around the world gathered in San Antonio, Texas for the 2011 AMDA/IPA Pompe Patient and Scientific Conference. Over the course of this weekend,...
