January 31, 2011
AMDA Webinar on Genzyme’s Next Generation Pompe Treatment: Clinical Development Overview
The AMDA is hosting yet another webinar for the Pompe community on Feb 3, 2011. Title: Genzyme’s Next Generation Pompe
CAMBRIDGE, Mass.—Genzyme Corp. today announced that it will build an additional manufacturing plant in Geel, Belgium, to support the long-term
Jan. 19, 2011—BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has initiated a Phase 1/2 trial for BMN 701,
January 1, 2011
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
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 LumizymeA form of enzyme replacement therapy used to treat Pompe disease by providing a lab-made version of the GAA enzyme. 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