Sentences with phrase «hemophilia b»

Heart disease (patent ductus arteriosus) occurs in Bichons, and a serious blood - clotting disease (hemophilia B).

Blood - clotting diseases (von Willebrand's, hemophilia A, hemophilia B) are potential concerns in the breed.

Blood - clotting diseases include hemophilia B, von Willebrand's, and thrombocytopenia.

Factor IX deficiency or hemophilia B: same as hemophilia A, but more rare and involves a different clotting factor.

Blood - clotting diseases include hemophilia B, factor VII deficiency, and von Willebrand's disease.

Cataracts and loosening of the retina are other ocular problems that have been seen in this breed as well as hemophilia B, osteo dystrophy, hypoglycemia, dwarfism, metabolic hepatic defect, osteo sarcoma, and diabetes.

(Queen Victoria passed the mutation for hemophilia B on to her son Leopold.)

However, they say the work at least proves the potential for mRNA therapy to successfully treat not only hemophilia B but also other human disorders, such as hemophilia A (caused by faulty clotting factor VIII) or a variety of diseases of the liver, central nervous system, lung and eyes.

Years after receiving a single dose of gene therapy, patients on the hemophilia B trial continued to produce their own clotting factor from the normal transferred gene with minimal side effects.

Good manufacturing practice production of self - complementary serotype 8 AAV vector for a hemophilia B clinical trial.

Concerns about immunological consequences of rAAV - mediated gene transfer were substantiated by the outcome of a clinical trial in which human hemophilia B patients were infused into the liver with rAAV -2-F.

Dr. High led the Center in the conduct of successful clinical studies of AAV - mediated gene therapy for a rare blinding condition and for hemophilia B.

Sangamo is also testing the liver - factory gene treatment in trials now getting underway for the blood - clotting disorder hemophilia B and another metabolic disease, Hurler syndrome.

Peppas and Horava's work has been patented and builds upon their published and patented system for the oral delivery of human factor IX (hFIX), a prophylactic treatment for hemophilia B patients.

«While an oral delivery platform will be beneficial to all hemophilia B patients, patients in developing countries will benefit the most,» said Sarena Horava, the study's lead author who is a recent Ph.D. graduate from the Cockrell School's McKetta Department of Chemical Engineering and a National Science Foundation graduate research fellow.

The researchers designed the oral delivery system, which contains micro - and nanoparticles, to carry a protein therapy that treats hemophilia B.

Formerly a research leader at CHOP, High pursued groundbreaking preclinical investigations in hemophilia B gene therapy and provided scientific expertise to previous gene therapy trials in hemophilia and other genetic disorders at CHOP before moving to Spark Therapeutics, which was spun off from CHOP in 2013.

Researchers are reporting the highest and most sustained levels to date of an essential blood - clotting factor IX in patients with the inherited bleeding disorder hemophilia B.

«After one dose of gene therapy, hemophilia B patients maintain near - normal levels of clotting factor.»

It was hemophilia B, which affects about 1 in 25,000 males in the U.S. — that's five times fewer sufferers than hemophilia A.

Most of this research has centered on hemophilia B, although hemophilia A is five times as common.

Patients with hemophilia A have a deficiency of clotting factor VIII (FVIII), while hemophilia B patients are deficient in clotting factor IX (FIX).

Not exact matches

Endari, the first new treatment for patients with sickle cell disease in almost 20 years, Genentech's Hemlibra, the first - ever non-blood product to treat patients with hemophilia A with inhibitors, Actemra, the first treatment for adults diagnosed with giant cell arteritis, BioMarin's Brineura, the first treatment for a form of Batten disease, Benznidazole, the first U.S. treatment for Chagas disease, Novartis» Kymriah to treat certain children and young adults with B - cell acute lymphoblastic leukemia, which is also the first gene therapy to become available in the United States, are some of the drugs that received the FDA's stamp of approval in 2017.