An update by Genzyme regarding treatment for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. Q: What is the status of the Pompe program? Genzyme has made strong progress within its Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More program since its decision earlier this year to move forward with an internally developed product...

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,...

In July 2002, Genzyme began the transition of the nine transgenic (rabbit derived enzymeA protein that helps the body carry out chemical reactions. More) patients to the CHO derived product. Three of the nine patients (infantile patients in the Netherlands) will remain on the transgenic enzymeA protein that helps the body carry out chemical reactions. More until April 2003, when supply will be...

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...

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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. Genzyme has invested significant resources over the past four years pursuing the most promising...

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...

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,...

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...

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...

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...

Article on Transgenic EnzymeA protein that helps the body carry out chemical reactions. More Replacement Therapy EnzymeA protein that helps the body carry out chemical reactions. More therapy for pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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...

This is an approximate transcript (i.e. from notes, not a tape) of an interview between Novazyme and the International Pompe Association (IPA). The participants were, from Novazyme: John Crowley (CEO), Julie Anne Smith (Senior Director, Patient Advocacy), William...

The following is a transcript of a telephone interview conducted for the IPA(International Pompe Association) by Kevin O’Donnell of the AGSD-UK (Association for GlycogenA stored form of sugar used for energy. More Storage Disease-United Kingdom. Kevin O’Donnell interviewed Paul Kaplan, General Manager of the...

Excerpt from Pharming Press Release—March 30, 2001 LEIDEN, the Netherlands, March 30, 2001 - Pharming Group N.V. announced the start of a Phase II-III clinical trialA research study that tests new treatments or approaches in people. More of an enzymeA protein that helps the body carry out chemical reactions. More replacement therapy for Pompe’s disease. The trial, under management of the...

Excerpt From Press Release-March 20, 2001 CAMBRIDGE, MA and LEIDEN,the Netherlands—Genzyme General (Nasdaq: GENZ), a division of Genzyme Corp., and Pharming Group N.V. (AEX: PHAR and EASDAQ: PHAR) announced the publication March 20 in Genetics in Medicine...

The following is a transcript of a telephone interview conducted for the IPA(International Pompe Association) by Kevin O’Donnell of the AGSD-UK (Association for GlycogenA stored form of sugar used for energy. More Storage Disease-United Kingdom. Kevin O’Donnell interviewed Paul Kaplan, General Manager of the...

Excerpt From Press Release—October 5, 2000 Genzyme General and Pharming Group N.V., announced today that results from the first two clinical trialsA research study that tests new treatments or approaches in people. More ever conducted for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More were presented this week at the American Society of Human Genetics (ASHG) meeting in...

Chen focused on the phase I/II trials underway at Duke with 3 IIa infants. Average age at diagnosis is 5 months and survival thereafter is 3 1/2 months on average, with a range of 0-9 months. Very few baby’s survive a year. Recombinant enzymeA protein that helps the body carry out chemical reactions. More from CHO cells (5 mg/kg...