Latest Inborn errors of metabolism Stories
NOVATO, Calif., Aug. 30 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has received orphan drug designation from the U.S.
NOVATO, Calif., Aug. 17 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) announced today that it has acquired ZyStor Therapeutics, Inc.
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.
The degradation of proteins and other macromolecules in cells is vital to survival.
Commonalities between flies and humans make a valuable new model for galactosemia.
SILVER SPRING, Md., May 25 /PRNewswire-USNewswire/ -- The U.S.
CAMBRIDGE, Massachusetts, May 19, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced that it has received the 2010 Partners in Progress Corporate Award from NORD.
In findings that advance scientists' understanding of a whole class of inherited disorders, a team from The Scripps Research Institute has shed light on a mechanism that enables a potential treatment for Gaucher's disease and other lysosomal storage diseases.
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.