Latest Glycogen storage disease type II Stories
CRANBURY, N.J., Aug.
The first commercially available treatment in the United States for patients with late-onset Pompe disease was administered today (Wednesday, June 16) at the University of Florida.
SILVER SPRING, Md., May 25 /PRNewswire-USNewswire/ -- The U.S.
SILVER SPRING, Md., May 24 /PRNewswire-USNewswire/ -- Genzyme Corp.
CRANBURY, N.J., May 6 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced financial results for the quarter ended March 31, 2010 and reviewed the progress on its product development pipeline including an update that the Company does not plan to advance AT2220, its investigational drug AT2220 (1-deoxynojirimycin HCl) for the treatment of Pompe disease, as a monotherapy at this time.
MONTREAL, March 29 /PRNewswire/ -- Enobia Pharma Inc. unveiled findings from the first hypophosphatasia (HPP) self-reported patient survey intended to evaluate the burden of illness associated with HPP at the 2010 American College of Medical Genetics (ACMG) Annual Clinical Genetics Meeting.
NEW YORK, Feb.
CRANBURY, N.J., Feb.
The HCMA Says a Simple Genetic Test Can Tell For Sure HIBERNIA, N.J., Feb. 1 /PRNewswire-USNewswire/ -- Hypertrophic Cardiomyopathy (HCM) is a genetic disease that causes the heart muscle to become abnormally large.
As scientists work to find new treatments for Pompe disease -- the devastating genetic "villain" that drives the efforts of the main characters in the new film "Extraordinary Measures" -- University of Florida researchers are hopeful that gene therapy will help patients in the late stages of the disease breathe on their own.
- One of the side scenes of the stage in a theater, or the space included between the side scenes.
- The outside stock exchange, or “curb market,” of Paris.
- A flute or groove on the blade of a sword.
- A section of stage scenery placed in a wing of a theatre.