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 MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More 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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) in the United States. MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More has been approved for the treatment of patients with Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More, a debilitating,...
European Commission Approves Genzyme’s Myozyme®
Product is First Treatment for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More Genzyme Corp. (Nasdaq: GENZ) announced today that it has received marketing authorization for Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More) in the European Union. MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More has been approved for long-term enzymeA protein that helps the body carry out chemical reactions. More 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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More). 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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More). If approved, MyozymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More 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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More” 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. More. 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. More of Myozyme® (alglucosidase alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More), which is being studied for the treatment of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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 alfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of ... More), an investigational enzymeA protein that helps the body carry out chemical reactions. More replacement therapy for Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More. 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 trialA research study that tests new treatments or approaches in people. More evaluating the use of its MyozymeTM recombinant human acid alpha-glucosidase enzymeA protein that helps the body carry out chemical reactions. More (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 diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More 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 trialA research study that tests new treatments or approaches in people. More evaluating the use of its Myozyme™ recombinant human acid alpha -Glucosidase enzymeA protein that helps the body carry out chemical reactions. More as a potential treatment for Pompe...