Genzyme to Build Additional Plant to Support Growth of Myozyme® and Lumizyme®
CAMBRIDGE, Mass.—Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term growth of Myozyme® and Lumizyme® for Pompe disease. The company held a ceremony today in Geel to mark the start of...
BioMarin Initiates Phase 1/2 Trial for BMN 701 for the Treatment of Pompe Disease
Jan. 19, 2011—BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a Phase 1/2 trial for BMN 701, a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA) in development for the treatment of Pompe...
2010 AMDA Research Grant Awarded to Dr. Andrea Amalfitano
In 2010 the AMDA solicited grant applications from researchers around the world. After careful consideration and consultation with our Scientific Advisory Board, Dr. Amalfitano of Michigan State University was selected as the recipient of the First Annual AMDA...
BMN-701: Potentially More Effective Treatment Option For Pompe
Title: BMN-701: Potentially more effective treatment option for Pompe
Date: Monday, December 13, 2010
Speaker:
Barbara Wuebbels, BioMarin
Jon Lebowitz, BioMarin
Diaphragm Delivery Of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase
Title: Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene Vector in Patients With Pompe Disease
Date: Tuesday, October 26, 2010
Speaker:
Barry J Byrne, MD, PhD – University of Florida
Autophagy and its Role in Pompe Disease
Title: Autophagy and it’s Role in Pompe Disease
Date: Wednesday, August 4, 2010
Speakers:
Nina Raben, MD, PhD – Staff Scientist, Arthritis + Rheumatism Branch, National Institute of Arthritis + Musculoskeletal + Skin Diseases (NIAMS), National Institutes of Health, Bethesda, MD.]
Pompe Patient Meeting Hosted by Columbia University
Dr. Maryam Banikazemi of Columbia University is hosting a meeting for Pompe patients on July 16, 2010 in New York, New York. The expert panel of speakers will include Drs. J. Bach, A. Slonim, T. Goldberg, and M. Banikazemi. For more details, please download meeting...
Pompe Program Update—FDA Approves Lumizyme
On May 24, 2010 the FDA approved Genzyme’s Lumizyme for the treatment of patients with Pompe disease who are over 8 years of age and do not have evidence of cardiac hypertrophy. For more information on the approval and how to access treatment, please read the Pompe...
The FDA Approves Lumizyme for Late-Onset Pompe Disease
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has granted U.S. marketing approval for LumizymeTM (alglucosidase alfa), produced at the 4000 liter (L) bioreactor scale at its manufacturing facility in Geel, Belgium. Lumizyme is the...
AMDA nominates Drs. Reuser and van der Ploeg to Rare Disease Day Hall of Fame
By: Tiffany House--AMDA President posted on: March 01, 2010 The AMDA has nominated Dr. Arnold Reuser and Dr. Ans van der Ploeg to the Rare Disease Day Research Hall of Fame. Drs. Reuser and van der Ploeg have spent decades researching Pompe disease, and from their...
Editorial: Extraordinary Measures or an Extraordinary Tale? (Update)
By: Tiffany House--AMDA President posted on: February 15, 2010 What happened to the TRUTH? In the wake of the recent article by Quest Magazine (January 2010) and the upcoming film “Extraordinary Measures” I feel that I must say something as to the accuracy of the...
Pompe Disease: The Real Story
To read a factual account of the true history of Pompe Disease, please visit Pompe Disease—The Real Story.
AMDA Press Release Regarding Extraordinary Measures
The AMDA issued a press release regarding the film, Extraordinary Measures. The press release can be found here. In addition, the AMDA created a one-page fact sheet regarding Pompe disease to help raise awareness of the condition. That fact sheet can be found...
ATAP Inclusion/Exclusion and List of Study Sites
This information was complied from the information provided on clinicaltrials.gov. To read, please download file by clicking here.
December 2009 Pompe Program Update
Genzyme would like to provide an update to the US Pompe community about the status of the Alglucosidase Alfa Temporary Access Program (ATAP) and the application for Lumizyme™ (alglucosidase alfa) approval in the United States. In a press release dated December 3,...
Pompe Disease—By a Pompe Patient
By Dawn Harris Kendall posted on: December 01, 2009 Living with Pompe Elizabeth Nunnery takes her infant daughter, Zoe, to the hospital at 8:30 in the morning. A nurse verifies Zoe’s medical information and hooks Zoe up to machines monitoring her blood oxygen level,...
Influenza Vaccinations and Pompe Disease
Guidelines from Erasmus Medical Centre, Rotterdam, the Netherlands As a guideline we recommend that all patients with Pompe disease are vaccinated against influenza. This applies both to the seasonal influenza as well as for the 2009 flu pandemic (influenza virus...
Genzyme invites the Gaucher, Fabry, Pompe and MPS I communities to a Town Hall Meeting
Genzyme Manufacturing Update Genzyme invites all patients to participate in a forum to discuss recent FDA reports and media attention related to manufacturing operations in Allston, Massachusetts. Date: Monday, November 23, 2009 Time: 6:00 PM – 7:00 PM (EST) US Toll...
November 2009 Pompe Program Update from Genzyme
Genzyme would like to provide an update to the US Pompe community about the status of the application for Lumizyme™ (alglucosidase alfa) produced at the 2000 L scale in the United States. To read full Program Update, please click here.
Amicus Therapeutics Announces Plan to Initiate Phase 1 Study of AT2220 for Pompe Disease
CRANBURY, N.J., Sept 30, 2009 - - Amicus Therapeutics today announced it plans to initiate a Phase 1 study of AT2220 (1-deoxynojirimycin HCl), its investigational drug in development for the treatment of Pompe Disease. The primary objective of this study is to...