Latest Thalassemia Stories

EAST HANOVER, N.J., January 23, 2013 /PRNewswire/ -- Novartis announced today that the US Food and Drug Administration (FDA) has approved Exjade(®) (deferasirox) for the treatment of chronic iron overload in patients 10 years of age and older with non-transfusion-dependent thalassemia (NTDT) syndromes and with a liver iron concentration of at least 5 mg of iron per gram dry weight and a serum ferritin measurement greater than 300 micrograms per liter. Exjade is the first...

TORRANCE, Calif., Dec. 3, 2012 /PRNewswire/ -- Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine company, today announced the completion of patient enrollment for its phase 3 clinical trial to study L-glutamine as a treatment for sickle cell disease. "The completion of enrollment is a significant milestone for our company and the sickle cell community, moving one step closer to potentially bringing the first widely available treatment to patients...

AMSTERDAM, June 15, 2012 /PRNewswire/ -- "There is a large potential opportunity for saving lives by early identification of cardiac iron loading in Thalassemia patients", declares Professor John-Paul Carpenter of the Royal Brompton CMR Unit in London at the 17th Congress of the European Hematology Association in Amsterdam. Thalassemia major (TM) is a substantial health issue, with over 25,000 new transfusion-dependent children identified each year around the world. In...

Researchers say their method, tested in human cells, may offer the first viable approach to gene transfer in sickle cell anemia A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment. This study's findings, published in PLoS ONE, represents a new approach to...

SAN DIEGO, March 12, 2012 /PRNewswire/ -- HemaQuest Pharmaceuticals, Inc. (HemaQuest), a biotechnology company focused on developing small molecule therapeutics to treat hemoglobin disorders, announced today that it has closed a $13 million extension of their Series B financing. The Series B Extension is being funded by its existing investors: Aberdare Ventures, De Novo Ventures, Forward Ventures, Latterell Venture Partners and Lilly Ventures. Proceeds from the financing will be...

BETHESDA, Md., Feb. 24, 2012 /PRNewswire-USNewswire/ -- During the second annual ACMG Foundation Day of Caring, the ACMG Foundation (www.acmgfoundation.org), a prominent non-profit genetics foundation based in Bethesda, MD will give new bicycles and helmets to 20 children from several North Carolina genetic disease support agencies at 10:00 am in Exhibition Hall C of the Charlotte, NC Convention Center. (Photo: http://photos.prnewswire.com/prnh/20120224/DC59195) Genetics...

EAST HANOVER, N.J., Dec. 13, 2011 /PRNewswire/ -- Results from THALASSA, the first pivotal placebo-controlled study examining the benefit of iron chelation with Exjade(®) (deferasirox) in patients with non-transfusion-dependent thalassemia (NTDT), show that Exjade can significantly reduce iron overload. These data were presented today at the 53rd Annual Meeting of the American Society of Hematology in San Diego. THALASSA investigated whether patients with NTDT and iron...

Research assessing the safety and efficacy of hydroxyurea therapy in pediatric patients with sickle cell disease (SCD) and the use of pre-operative transfusions for patients with SCD who undergo low- and moderate-risk elective surgery will be presented today at the 53rd Annual Meeting of the American Society of Hematology. An estimated 90,000 to 100,000 Americans are affected each year by SCD, a serious disorder that causes normal red blood cells to become rigid and form in a crescent...

SAN DIEGO, Dec. 11, 2011 /PRNewswire-USNewswire/ -- Research assessing the safety and efficacy of hydroxyurea therapy in pediatric patients with sickle cell disease (SCD) and the use of pre-operative transfusions for patients with SCD who undergo low- and moderate-risk elective surgery will be presented today at the 53rd Annual Meeting of the American Society of Hematology. An estimated 90,000 to 100,000 Americans are affected each year by SCD, a serious disorder that causes normal...