As noted, another limitation is that the 12-month study period was too short to adequately capture improvements in pediatric-specific parameters such as puberty (as evaluated
by Tanner stage) and bone mineral density analysis. However, these parameters will continue to be followed in extension study PB-06-006 (NCT01411228) that will capture an additional 2 years Transmembrane Transporters activator of data for a total of 3 years of taliglucerase alfa treatment. In summary, this report demonstrates that taliglucerase alfa improves the hematologic and visceral manifestations of Gaucher disease in children. It broadens the findings to date of the safety and efficacy of taliglucerase alfa in patients with GD, pediatric and adult patients alike, and as such expands the potential treatment options for management of this genetic metabolic disorder. AZ designed the study, performed research, analyzed data, and wrote the paper; DEG-R performed research and wrote the paper; AA performed research and wrote the paper; DE assisted
this website with the research and wrote the paper; AP designed the study, analyzed and verified data, and wrote the paper; EB-A designed the study, analyzed and verified data, and wrote the paper; and RC designed the study, analyzed and verified data, and wrote the paper. None of the authors received compensation for their contributions to this manuscript. AZ receives consultancy fees from and Diflunisal has stock options in Protalix BioTherapeutics and is a member of their Scientific Advisory Board. In addition, AZ receives support from Genzyme for participation in the International Collaborative Gaucher Group Registry, and receives honoraria from Shire HGT, Actelion, and Pfizer; DEG-R and AA are study investigators; DE has received honoraria from and had travel/accommodation expenses covered/reimbursed by Shire HGT and Pfizer. In addition, the Gaucher Clinic, for which DE is the site coordinator, has had clinical trial expenses reimbursed; AP, EB-A, and RC are employees of Protalix BioTherapeutics. The authors would like to acknowledge fellow
investigator and pediatrician Dr. Rene Heitner from Johannesburg, South Africa, who passed away in January 2012. The authors would also like to acknowledge Dr. Peter Cooper of Johannesburg, South Africa, who is treating Dr. Rene Heitner’s patients in study PB-06-006, the taliglucerase alfa pediatric extension trial. This study was sponsored by Protalix BioTherapeutics. Editorial and medical writing support was provided by Elizabeth Daro-Kaftan, PhD, of Peloton Advantage, LLC, and was funded by Pfizer. Pfizer and Protalix entered into an agreement in November 2009 to develop and commercialize taliglucerase alfa. “
“Acute Myeloid Leukemia (AML) is primarily a hematological malignancy of the elderly with a median age of onset at 60 years and a poor prognosis with a five year survival rate of only 12% .