By: Erica Dimitropoulos Co-Copy Editor [Content- Focused]  – On February 14, 2014, elosulfase alfa (Vimizim™) became an FDA-approved enzyme replacement therapy for Morquio A Syndrome, a type of mucopolysaccharidosis (MPS IVA).1 Elosulfase alfa was granted priority review and was also the first drug to receive the Rare Pediatric Disease Priority Review Voucher that motivates the development…

By: Tamara Yunusova, Senior Staff Editor – Approximately 3.2 million Americans have chronic hepatitis C infection.1 While acute cases are not common, rates of chronic hepatitis C continue to surge due to the recent discovery of the virus in 1989 and consequently, the establishment of a test screening for HCV antibodies in 1992.1 HCV has…

By: Neal Shah, Co Editor-In-Chief – Pure Red Cell Aplasia (PRCA), also known as erythroblastopenia, is characterized by a suppression of erythrocytes in the bone marrow.  It is a peculiar oddity that the bone marrow’s progenitor cells still differentiate into white blood cells and platelets.1  PRCA has idiopathic, viral, auto-immune, and genetic etiologies.  Diamond-Blackfan syndrome…

By: Khilna Patel, Pharm.D. Candidate c/o 2012 – On October 25 of this year, Eli Lilly and Company announced a worldwide market withdrawal of Xigris (drotrecogin alfa), a drug previously indicated to treat severe sepsis in high-risk patients.  Drotrecogin alfa is a recombinant form of human activated protein C.  The efficacy of drotrecogin alfa was…