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. Gaucher disease occurs in people who do not produce enough of an enzyme called glucocerebrosidase. Without this enzyme, harmful amounts of a certain fatty substance (lipid) can build up in the liver, spleen, bones, bone marrow and nervous system, and can prevent cells and organs from working properly. About 1 in 50,000 to 1 in 100,000 people in the general population have Gaucher disease…

Amicus Therapeutics (Nasdaq: FOLD) announced additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in Miami, Florida. The Company also presented encouraging data from preclinical studies evaluating the combination of pharmacological chaperones and enzyme replacement therapy (ERT) for Fabry disease and Pompe disease as well as from preclinical studies examining the use of pharmacological chaperones for the treatment of Parkinson’s disease…

Protalix BioTherapeutics, Inc. (NYSE-Amex: PLX), announced that additional data from the Company’s pivotal Phase III clinical trial of taliglucerase alfa in patients with Gaucher disease was presented at the Annual Meeting of the Lysosomal Disease Network: WORLD Symposium 2010 in Miami, Florida, during an oral session titled, “Novel Enzyme Replacement Therapy for Gaucher Disease: Phase III Pivotal Clinical Trial with Plant Cell Expressed Recombinant Glucocerebrosidase (prGCD) - taliglucerase alfa.” The oral presentation was made by Hanna Rosenbaum M.D…

Preliminary investigations by US researchers suggest that a vaccine made with leukemia cells appears able to reduce or wipe out the last few cancer cells that are left behind in some patients with chronic myeloid leukemia (CML) who are taking the drug Gleevec (Imatinib mesylate). However, the researchers said the results are tentative and there could be other reasons for this apparent success…

UroToday.com - Ours is a retrospective review of patients who underwent open retroperitoneal lymph node dissection between 2001-2008. We identified perioperative data for patients who underwent primary (P-RPLND) versus post-chemotherapy RPLND (PC-RPLND) and found mean blood loss, operative duration and hospital stay to be significantly less for the former group (P This contemporary data should be considered when comparing open versus laparoscopic RPLND (L-RPLND)…