Latest Glucocerebrosidase Stories

Pathway disrupted in Gaucher disease affects Parkinson's-associated protein deposits, suggests novel therapeutic strategyMassachusetts General Hospital investigators appear to have found the mechanism behind a previously reported link between the rare genetic condition Gaucher disease and the common neurodegenerative disorder Parkinson's disease. In a report to appear in the July 8 issue of Cell and receiving early online release, they describe how disruption of the molecular pathway that...

As Parkinson's Awareness Month gets underway, a Canadian-led international study is providing important new insight into Parkinson's disease and paving the way for new avenues for clinical trials. The study, led by Dr. Michael Schlossmacher in Ottawa, provides the first link between the most common genetic risk factor for Parkinson's and the hallmark accumulation of a protein called alpha-synuclein within the brains of people with Parkinson's. It is published in the most recent edition of the...

CAMBRIDGE, Mass., Nov. 4, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, Nasdaq: SHPGY), the global specialty biopharmaceutical company, today presented positive new data from a Phase III clinical trial (study 039) designed to evaluate the efficacy of VPRIV® (velaglucerase alfa for injection) compared with imiglucerase in patients with type 1 Gaucher disease at the 2010 Annual American Society of Human Genetics (ASHG) in Washington, D.C. The study met its primary endpoint and adds...

CAMBRIDGE, Massachusetts, August 26, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the European Commission has granted marketing authorisation for VPRIV(R) (velaglucerase alfa), a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of type 1 Gaucher disease. VPRIV has been authorized as an orphan medicine through the Centralised Procedure, making it available in 30...

CAMBRIDGE, Massachusetts, June 25, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency adopted a positive opinion on the marketing authorisation for VPRIV(TM) (velaglucerase alfa), the company's enzyme replacement therapy (ERT) for the treatment of type 1 Gaucher disease. The CHMP positive opinion will now be...

The new findings may inspire clinical trialsIn 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.The findings were published in an advance, online edition of the journal Nature Chemical Biology on May 9, 2010."This study is likely to motivate clinical trials for the treatment of...

CAMBRIDGE, Massachusetts, February 26, 2010 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease in pediatric and adult patients. The FDA designated VPRIV for Priority Review and granted marketing approval in just...

New drug will offer a treatment alternative for patients with rare genetic disorder SILVER SPRING, Md., Feb. 26 /PRNewswire-USNewswire/ -- The U.S. Food and Drug Administration has approved velaglucerase alfa for injection (VPRIV) to treat children and adults with a form of the rare genetic disorder Gaucher disease. (Logo: http://www.newscom.com/cgi-bin/prnh/20090824/FDALOGO) Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this...

DUBLIN, November 24 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced that it has submitted a MAA to the European Medicines Agency for velaglucerase alfa, the company's enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease. This is the third marketing application for velaglucerase alfa that has been submitted, with previous submissions in the United States and Canada. Based...

CAMBRIDGE, Massachusetts, November 4 /PRNewswire-FirstCall/ -- Shire plc (LSE: SHP, NASDAQ: SHPGY), the global specialty biopharmaceutical company, today announced that the United States Food and Drug Administration (FDA) has granted Priority Review for the New Drug Application (NDA) for velaglucerase alfa, the company's enzyme replacement therapy in development for the treatment of Type 1 Gaucher disease. Priority Review designation is given to drugs that offer major advances in...