Latest enzyme replacement therapy Stories
DOYLESTOWN, Pa., May 14, 2013 /PRNewswire/ -- Callidus Biopharma, Inc., a development-stage biotechnology company focused on creating breakthrough biologic drugs for a range of orphan diseases, announced today that it has closed on $4.6 million in Series A financing led by two strategic investors. The Company intends to use the proceeds to accelerate pre-clinical development of its drug discovery pipeline of therapies for lysosomal storage diseases (LSDs), including lead candidates...
LEXINGTON, Massachusetts, February 19, 2013 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPG), announces that the Lysosomal Disease Network (LDN) presented Shire Human Genetic Therapies (HGT) with the first ever Novel Treatment Award for VPRIV on February 14th, at its 9th Annual WORLD Symposium. The LDN have launched this new award, which will be presented annually, to recognize new drugs or therapies that have made a considerable contribution in the area of lysosomal...
NYON, Switzerland, June 28, 2012 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today presented new data that show VPRIV(R) (velaglucerase alfa for injection), the company's enzyme replacement therapy for type 1 Gaucher disease, significantly improved selected markers of Gaucher-related bone disease in patients. These data were presented at the European Working Group on Gaucher Disease (EWGGD) meeting held in Paris, France,...
NOVATO, Calif., Feb. 28, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced that the FDA Office of Orphan Products Development has granted orphan drug designation for UX003 for the treatment of MPS 7. MPS 7 is an extremely rare autosomal recessive lysosomal storage disorder characterized by a deficiency of the lysosomal enzyme Beta-glucuronidase, required for the...
LEXINGTON, Massachusetts, February 22, 2012 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use has approved the production of VPRIV(R) (velaglucerase alfa) in its new state-of-the-art manufacturing facility at 400 Shire Way in Lexington, MA. The European Commission's decision is expected imminently. "We welcome the...
A team of scientists, led by researchers at the University of California, San Diego School of Medicine and Zacharon Pharmaceuticals, have developed a simple, reliable test for identifying biomarkers for mucopolysaccharidoses (MPS), a group of inherited metabolic disorders that are currently diagnosed in patients only after symptoms have become serious and the damage possibly irreversible. The findings will be published online January 8 in the journal Nature Chemical Biology. MPS is...
Kids with Pompe disease fail because of a missing enzyme, GAA, that leads to dangerous sugar build-up, which affects muscles and movement. An enzyme replacement treatment pioneered at Duke University has saved many lives, but some children with Pompe disease produce an immune reaction that blocks the benefits of the life-saving enzyme treatment. To date there has been no success in eliminating or suppressing this immune response. Now research led by Duke University Medical Center, with...
NOVATO, Calif., Jan. 5, 2012 /PRNewswire/ -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced it has in-licensed an enzyme replacement therapy program from St. Louis University to treat mucopolysaccharidosis type 7 (MPS 7). The in-licensed program is a treatment for an ultra-rare genetic, metabolic disorder that results from the deficiency of the...
CARMIEL, Israel, Oct. 26, 2011 /PRNewswire/ -- Protalix BioTherapeutics, Inc. (NYSE-AMEX: PLX, TASE: PLX), today announced that senior management will present at four upcoming conferences: Ernst & Young Journey 2011 Conference Wednesday, November 2, 2011 at 2:45 PM IDTTopic: The Pfizer-Protalix Gaucher Collaboration - The Partners' Point of ViewDr. David Aviezer, President & CEOHilton Hotel, Tel-Aviv, Israel PDA-Parenteral Drug Association / FDA-Food and Drug Administration:...
NYON, Switzerland, Aug. 31, 2011 /PRNewswire/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced positive data from several studies designed to evaluate the safety of switching to REPLAGAL (agalsidase alfa) from Fabrazyme® (agalsidase beta) and the long-term safety and benefits of switching to VPRIV (velaglucerase alfa) from Cerezyme® (imiglucerase). Study results are being presented at the 2011 Annual Symposium of the Society for...
