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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme.), an investigational enzymeA protein that helps the body carry out chemical reactions. replacement therapy for 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.. 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...
IPA/Genzyme Meeting
Board members of the IPA, the International Pompe Association, met with key representatives of Genzyme in Cambridge, Massachusetts on April 16-17, 2002. The IPA members in attendance were: Randall House, AMDA-US; Marylyn House, AMDA-US, Ria Broekgaarden, VSN-The...
Genzyme Press Release
Excerpt From Genzyme’s April 17, 2002 Press Release Pompe Update Genzyme is moving forward aggressively with its program to develop an effective therapy for Pompe disease. Genzyme has invested significant resources over the past four years pursuing the most promising...
Letter from Vice President of Genzyme
Following is a letter sent to the IPA by Jan van Heek, Executive Vice President of Genzyme, for release prior to the VSN Pompe Patient Conference which took place on March 9, 2002. in the Netherlands. Dear Friends, On Saturday, March 9th, 2002, I will be speaking at...
IPA Board Meeting – February 18-19, 2002
The first official IPA Board Meeting occurred in Rotterdam, The Netherlands on Feb. 18-19, 2002. Future agendas were planned and the following committees were formed. Communications—Kevin O’Donnell, Alan Muir, Marylyn House Patient Organizations—Maryze Schoneveld,...
Letter from IPA Chairman to Pompe Patients
The International Pompe Association (IPA) board will meet on February 18-19th, 2002. Representatives of scientific/medical teams and Genzyme will attend. The following issues will be discussed: 1. Finalize the universal patient questionnaire to be distributed in the...
Genzyme Acquires Manufacturing Facility in Europe
October 2001—CAMBRIDGE, Mass.—Genzyme Corporation announced today that it has acquired certain assets of Pharming N.V., the Belgian subsidiary of the Pharming Group currently operating under a court-supervised receivership. These assets include a 70,000 square foot...
2001 Conference
The first AMDA Patient Conference was held in San Antonio, Texas on September 28-30, 2001. It was attended by approximately 100 participants from around the world. The agenda included presentations from doctors directly involved in current clinical trialsA research study that tests new treatments or approaches in people. for Acid...
Finding normal; Teen battles rare enzyme disorder—San Antonio Express News
By: M. Pisano posted on: September 24, 2001 (originally published in San Antonio Express-News on Sept. 24, 2001) When Tiffany House was growing up, she was often sick. But in that resilient way that children adapt to their bodies and their immediate world, she thought...
Genzyme to Acquire Novazyme Pharmaceuticals
Proprietary Glycoprotein Technology Platform Will Advance Lysosomal Storage Disorder Programs Excerpt from August 7, 2001 Press Release Genzyme Corp. and privately held Novazyme Pharmaceuticals Inc. today announced a definitive merger agreement under which Novazyme...
Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk
Article on Transgenic Enzyme Replacement Therapy Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk Van den Hout JM, Reuser AJ, de Klerk JB, Arts WF, Smeitink JA, Van der Ploeg AT. Department of Pediatrics, Sophia Children’s...