Showing 2919 results for "hemophilia"

Hemophilia B Treatment, Idelvion, Approved for Use in Japan

Japan’s Ministry of Health, Labor and Welfare has approved CSL Behring‘s Idelvion (albutrepenonacog alfa) for the prevention of bleeding tendency in patients with hemophilia B. Hemophilia B, a bleeding condition that mainly affects males, is characterized by a deficient or defective factor IX protein, and is treated with intravenous infusion of factor IX.

Rare New Case of Pseudotumor in non-Hemophiliac Patient Reported

Researchers at the Mayo Clinic Arizona have reported a third rare case of hemophilic pseudotumor (HP) identified in a non-hemophiliac patient. Findings from the investigation, “Hemophilic pseudotumor in a non-hemophilic patient treated with a hybrid procedure of preoperative embolization of the feeding arteries followed by surgical resection—A…

Type of Chromatin Seen to Stop ‘Jumping Genes’ from Doing Harm, as in Hemophilia

New research suggests that the main developmental role of heterochromatin, a form of dense DNA found in chromosomes, is to suppress virus-like DNA elements known as “jumping genes” from replicating and attaching themselves across a person’s genome, potentially destroying important genes and causing a variety of diseases, including hemophilia and cancer. The study, by University of North Carolina…

Sangamo’s Genome Editing Therapy for Hemophilia B Named ‘Orphan Drug’ by FDA

Sangamo BioSciences announced that its zinc finger nuclease (ZFN)-mediated genome editing product, SB-FIX, has received orphan drug designation from the U.S. Food and Drug Administration (FDA), speeding its development as a potential treatment for hemophilia B. Sangamo plans to initiate a Phase 1/2 clinical trial, SB-FIX-1501, in adults with this disease later…