December 13, 2010
BMN-701: Potentially More Effective Treatment Option For Pompe
Webinar Details Title: BMN-701: Potentially more effective treatment option for Pompe Date: Monday, December 13, 2010 Speaker: Barbara Wuebbels, BioMarinJon
Webinar Details Title: DiaphragmThe main muscle used for breathing. Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV-GAA) Gene VectorA delivery system, often based on a modified virus, used to carry genetic material into cells. in Patients With 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. Date:
August 4, 2010
Autophagy and its Role in Pompe Disease
Webinar Details Title: AutophagyThe cell's internal cleanup system that removes damaged or unneeded material. and it’s Role in Pompe Disease Date: Wednesday, August 4, 2010 Speaker:Nina Raben, MD, PhD – Staff
June 14, 2010
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,
May 27, 2010
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
CAMBRIDGE, Mass. – Genzyme Corporation (Nasdaq: GENZ) announced today that the FDA has granted U.S. marketing approval for LumizymeTM (alglucosidase
January 21, 2010
Pompe Disease: The Real Story
To read a factual account of the true history of Pompe Disease, please visit Pompe Disease—The Real Story.
January 20, 2010
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,
December 7, 2009
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 4, 2009
December 2009 Pompe Program Update
Genzyme would like to provide an update to the US Pompe community about the status of the Alglucosidase AlfaA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme. Temporary