Latest Inborn errors of metabolism Stories
CAMBRIDGE, Massachusetts, October 21 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces plans to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease, by the end of the year.
NOVATO, Calif., Aug. 19 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
Supplementing obese rats with the nutrient carnitine helps the animals to clear the extra sugar in their blood, something they had trouble doing on their own, researchers at Duke University Medical Center report.
Researchers from CIC bioGUNE's Structural Biology Unit and Columbia University (New York) have conducted a joint research project, published in the prestigious scientific journal Structure, to gain in-depth knowledge of the structure of pyruvate carboxylase when it is in solution
LEXINGTON, Massachusetts, July 16 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces it has received Fast Track designation from the U.S.
NOVATO, Calif., July 13 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc.
LEXINGTON, Massachusetts, July 6 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announces that, at the request of the FDA, in view of a potential restriction on the availability of the current approved and marketed treatment for Gaucher Disease patients, it has filed a treatment protocol for velaglucerase alfa, its enzyme replacement therapy in development for the treatment of Gaucher Disease. If approved by the FDA, the...
SOUTH SAN FRANCISCO, Calif., June 30 /PRNewswire/ -- Hyperion Therapeutics, a privately held specialty pharmaceutical company focused on the development of therapies that address critical unmet needs in the areas of gastroenterology and hepatology, today announced it has closed a $60 million Series C financing.
- FDA Agreement Reached on Phase 3 Study Design, Primary Endpoint and Histological Methodology - CRANBURY, N.J., June 22 /PRNewswire-FirstCall/ -- Amicus Therapeutics (Nasdaq: FOLD) today announced it has commenced the U.S.