Genzyme Pompe Program Update—October 2007
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MTAP Fact Sheet June 2007
MTAP, the Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—June 2007
Genzyme would like to take this opportunity to provide a brief update to the Pompe patient community regarding the US supply of Myozyme® (alglucosidase alfa). As planned, Genzyme submitted the necessary documentation to the FDA this month for the licensure of the...
MTAP Fact Sheet May 2007
MTAP, the Myozyme® (alglucosidase alfa) Temporary Access Program, is a clinical program to provide access to Myozyme produced by a larger scale manufacturing process for a limited time until this manufacturing scale is approved by the FDA. Patients enrolled in MTAP...
Genzyme Pompe Program Update—April 2007
As many of you are aware, a standard part of the biologics drug development process is to incrementally scale-up manufacturing capacity as demand for the product increases. During the development of Myozyme®, Genzyme began the manufacturing process using a smaller...
2006 Conference
2006 AMDA/IPA Conference Report By Allan Muir With contributions from George Fox and Jared Salbato Friday—November 17, 2006 Texas Hoe Down at Sea World Report by George Fox On Friday evening after check in and registration, the AMDA hosted a reception “Hoe Down” at...
S.A. conference to offer information on Pompe disease—San Antonio Express-News
By: Mariana Pisano posted on: November 14, 2006 (Published to web on November 14, 2006) Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup that damages muscles and can affect breathing and, in some cases, the heart., a rare, devastating and often fatal genetic disorder, is the subject of a major conference to be held Friday through Sunday at the Holiday Inn Riverwalk. To read...
AGSD (UK) Family Conference, October 16th 2005
Pompe Workshop Report Allan Muir, Pompe Representative, AGSD(UK) On October the 16th this year we held our annual AGSD (UK) family conference in Milton Keynes. For the Pompe Workshop we had a good number of families present and hopefully I left enough time in and...
Editorial: Review of “The Cure” by Geeta Anand
By: Marylyn House posted on: September 19, 2006 Dear All, I admit that I was wrong in my previous analysis of “The Cure:” an analysis which was based on summaries and reviews I found at Amazon.com. In fact, I must say that I was unexpectedly surprised while reading...
Editorial: Response to “The Cure” by Geeta Anand
By: Marylyn House posted on: September 02, 2006 (this response was written based on an expert found on Amazon.com and an article in the Wall Street Journal) Dear All, The following excerpt from Geeta Anand’s “story” on John Crowley can be found in the “Marketplace”...
Letter From IPA and Genzyme Concerning LOTS
Dear IPA members, Pompe patients, family members and GSD-net participants, The IPA board received several questions concerning participation in the LOTS trial. As there are certain restrictions to the market approval of Myozyme we asked Genzyme to make a written...
FDA Approves Genzyme’s Myozyme® for All Patients with Pompe Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that the Food and Drug Administration has granted marketing approval for Myozyme® (alglucosidase alfa) in the United States. Myozyme has been approved for the treatment of patients with Pompe disease, a debilitating,...
European Commission Approves Genzyme’s Myozyme®
Product is First Treatment for Pompe Disease Genzyme Corp. (Nasdaq: GENZ) announced today that it has received marketing authorization for Myozyme® (alglucosidase alfa) in the European Union. Myozyme has been approved for long-term enzymeA protein that helps the body carry out chemical reactions. replacement therapy in...
Genzyme Provides Update on U.S. Marketing Application for Myozyme
Genzyme Corp. (Nasdaq: GENZ) announced today that the Food and Drug Administration has extended by 90 days the review period for the biologics license application for Myozyme® (alglucosidase alfa). The new action date for the application is April 28, 2006. The purpose...
The IPA Pompe Connections Brochure
The IPA has developed the Pompe connections brochure to help get patients and their families timely and relevant information about Pompe disease and the developments that are happening. Please visit the Pompe Connections Brochure page.
Genzyme Submits BLA for Myozyme® for Treatment of Pompe Disease
Genzyme Corp. (Nasdaq: GENZ) announced today that it has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration for Myozyme® (alglucosidase alfa). If approved, Myozyme would be the first treatment developed for patients with Pompe...
Honors grad thrives on defying the odds—San Antonio Express-News
By: A. Dorsett posted on: May 13, 2005 (Originally published May 13, 2005) By doctor’s estimates, Tiffany House shouldn’t be alive today, more than 10 years after she was diagnosed with a rare, disabling genetic disease that robs its victims of muscle strength and,...
Summary of the 2005 AFM-IPA Conference
May 11th 2005 IPA Conference Pompe’s disease Nantes, France. “Emerging therapies for Pompe disease” Dr. Ans van der Ploeg, Erasmus MC Rotterdam the Netherlands, talked about the natural course in late onset Pompe diseaseA form of Pompe disease that begins after infancy and usually progresses more slowly.. She informed the audience about the outcome...
Genzyme Reports Interim Results from Pivotal Study of Myozyme
CAMBRIDGE, Mass.—Genzyme Corp. (Nasdaq: GENZ) announced today that it has completed a planned analysis of interim data from its pivotal clinical trialA research study that tests new treatments or approaches in people. of Myozyme® (alglucosidase alfa), which is being studied for the treatment of Pompe disease. The interim analysis was...
Anything for a Cure—People Magazine
By: By Richard Jerome. Macon Morehouse in San Antonio. posted on: March 21, 2005 Doctors Said Tiffany House Wouldn’t Live Past 20, but Her Parents Refused to Accept That Death Sentence (Published in People Magazine: March 21, 2005) When Randall and Marylyn House...