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 enzyme 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 disease. 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 trial 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...
Genzyme Files for European Approval of Myozyme® For Treatment of Pompe Disease
CAMBRIDGE, MA—Genzyme Corp. (Nasdaq: GENZ) announced today that the European Medicines Agency (EMEA) has accepted its marketing authorization application for Myozyme® (alglucosidase alfa), an investigational enzyme replacement therapy for Pompe disease. If approved,...
Summary of 2004 International Pompe Association Conference
On 9th and 10th October, the AGSD (UK) held its annual patient conference. This year we were more ambitious than usual in that the Pompe Workshop extended into a second day. Families and speakers arrived on the Friday night and we held an informal conference dinner on...
IPA Conference – October 30 – November 1, 2003
Medical experts and patients are invited to attend the International Pompe Conference 2003. The goal of the conference is to bring together scientific expertise and patients’ experience. On the way towards a therapy for Pompe’s Disease there were and are many examples...
Editorial: Is Transgenic Enzyme For Pompe’s Disease Really Dead?
By: Marylyn House posted on: June 03, 2003 Recently, I read an article in The Economist Technology Quarterly, December 14, 2002, entitled “Brave New World of Farmaceuticals” (British Spelling) which emphasized the need of more foundries to produce monoclonal proteins....
Genzyme Begins Enrolling Patients in Pivotal Pompe Disease Study
Genzyme General (Nasdaq: GENZ), a division of Genzyme Corp., announced today that it has treated the first patient in a pivotal clinical trial evaluating the use of its MyozymeTM recombinant human acid alpha-glucosidase enzyme (rhGAA) as a potential treatment for...
Editorial: Response to March 2003 Article in Quest
By: Marylyn House posted on: March 28, 2003 The following correspondence is in response to the Pompe disease research article that was published in the March 2003 issue of “Quest” an MDA publication. It includes emails sent over the GSDnet during February-March 2003,...
Genzyme General Begins Enrolling Patients in Pompe Disease Study
CAMBRIDGE, Mass – Genzyme General (Nasdaq: GENZ), a division of Genzyme Corp., announced today that it has begun enrolment in a clinical trial evaluating the use of its Myozyme™ recombinant human acid alpha -Glucosidase enzyme as a potential treatment for Pompe...
Genzyme’s Pompe Program Update
An update by Genzyme regarding treatment for Pompe disease. Q: What is the status of the Pompe program? Genzyme has made strong progress within its Pompe disease program since its decision earlier this year to move forward with an internally developed product...
IPA/Genzyme News—September 24, 2002
IBaarn, October 2nd 2002 Dear IPA members, Herewith I would like to inform you about the IPA/Genzyme telephone conference of September 24 2002. We discussed issues concerning the production, the transition, new trials and the meeting with the Erasmus University, VSN,...
Transgenic Enzyme Production to Cease
In July 2002, Genzyme began the transition of the nine transgenic (rabbit derived enzyme) patients to the CHO derived product. Three of the nine patients (infantile patients in the Netherlands) will remain on the transgenic enzyme until April 2003, when supply will be...