Blood Disorder News and Research

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Change in sickle cell anemia prevention strategies decreases racial disparities in stroke-related deaths

The disparity in stroke-related deaths among black and white children dramatically narrowed after prevention strategies changed to include ultrasound screening and chronic blood transfusions for children with sickle cell anemia, according to research presented at the American Stroke Association's International Stroke Conference 2012.

3 Feb 2012

Gene therapy can reduce painful bleeding episodes in patients with hemophilia B

Symptoms improved significantly in adults with the bleeding disorder hemophilia B following a single treatment with gene therapy developed by researchers at St. Jude Children's Research Hospital in Memphis and demonstrated to be safe in a clinical trial conducted at the University College London (UCL) in the U.K.

12 Dec 2011

FDA approves changes to REMS for Nplate and Promacta

The approval of changes to the Risk Evaluation and Mitigation Strategies (REMS) for both Nplate (romiplostim) and Promacta (eltrombopag) was announced by the U.S. Food and Drug Administration.

7 Dec 2011

EC extends therapeutic indication for Soliris to treat atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. today announced that the European Commission (EC) has extended the therapeutic indication for Soliris® (eculizumab) to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS).

29 Nov 2011

Almac collaborates with Alexion for packaging and distribution of Soliris during German E. coli outbreak

Almac, a leading provider of contract services to the global pharmaceutical and biotech industries, responded earlier this year to an urgent request from their client partner, Alexion Pharmaceuticals, Inc., a global biotechnology company, for packaging and distribution of Soliris (eculizumab) for patients with STEC-HUS during the E. coli outbreak in Germany earlier this year.

21 Nov 2011

Researchers discover new class of antimalarial compounds

Researchers have discovered a group of chemical compounds that might one day be developed into drugs that can treat malaria infection in both the liver and the bloodstream.

18 Nov 2011

Researchers to begin drug development projects for rare and neglected diseases

Researchers will begin drug development projects for rare and neglected diseases that include potential treatments for a musculoskeletal disorder, a cognitive dysfunction disorder, a virus that affects the central nervous system of newborns, a parasitic worm infection, a form of muscular dystrophy and a rare lung disease.

16 Nov 2011

Public health officials should address needs of people with blood disorders

Public health should focus not only on reducing the burden of common diseases but also address the needs of people with blood disorders , experts say in a supplement to December's American Journal of Preventive Medicine.

16 Nov 2011

Increasing TR2/TR4 expression can lead to higher fetal hemoglobin levels in sickle cell patients

A University of Michigan Health System laboratory study reveals a key trigger for producing normal red blood cells that could lead to a new treatment for those with sickle cell disease.

3 Nov 2011

Alexion third quarter total revenues increase to $204.0 million

Alexion Pharmaceuticals, Inc. today announced financial results for the three and nine months ended September 30, 2011.

20 Oct 2011

ApoPharma receives FDA approval for Ferriprox to treat transfusional iron overload

The U.S. Food and Drug Administration today approved Ferriprox (deferiprone) to treat patients with iron overload due to blood transfusions in patients with thalassemia, a genetic blood disorder that causes anemia, who had an inadequate response to prior chelation therapy.

15 Oct 2011

Potential new protein target for sickle cell disease

National Institutes of Health-funded scientists have corrected sickle cell disease in adult laboratory mice by activating production of a special blood component normally produced before, but not after, birth.

14 Oct 2011

Study identifies gene mutation responsible for higher frequency of Italian CDA II

Many of you might know that Congenital Dyserythropoietic Anemia type II (CDA II) is a rare blood disorder, due to a failure in final part of erythropoiesis. What will surprise you is the fact that some mutations responsible for the disease can be tracked 3.000 years back.

10 Oct 2011

Researchers use patient's own stem cells to correct genetic alteration that causes SCD

Using a patient's own stem cells, researchers at Johns Hopkins have corrected the genetic alteration that causes sickle cell disease (SCD), a painful, disabling inherited blood disorder that affects mostly African-Americans. The corrected stem cells were coaxed into immature red blood cells in a test tube that then turned on a normal version of the gene.

29 Sep 2011

Alexion receives FDA approval for Soliris to treat atypical Hemolytic Uremic Syndrome

The U.S. Food and Drug Administration today approved Soliris (eculizumab) to treat patients with atypical Hemolytic Uremic Syndrome (aHUS), a rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.

26 Sep 2011

EMA CHMP adopts positive opinion for Soliris to treat atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending that the therapeutic indication for Soliris® (eculizumab) be extended to include the treatment of pediatric and adult patients with atypical hemolytic uremic syndrome (aHUS) in Europe.

23 Sep 2011

ODAC recommends ApoPharma's Ferriprox for FDA approval to treat transfusional iron overload

ApoPharma Inc. today announced that the Oncologic Drugs Advisory Committee (ODAC) to the U.S. Food and Drug Administration (FDA) voted 10 - 2 to recommend that the FDA grant accelerated approval of Ferriprox (deferiprone), an oral iron chelator, for the treatment of patients with transfusional iron overload when current chelation therapy is inadequate.

15 Sep 2011

Encouraging results from Alexion's eculizumab Phase 2 study on generalized myasthenia gravis

Alexion Pharmaceuticals, Inc. today announced that the Company's study of eculizumab in a small group of 14 patients with severe and refractory generalized myasthenia gravis, an ultra-rare and debilitating form of generalized myasthenia gravis (gMG), showed a strong disease improvement signal.

15 Sep 2011

Enrollment complete in HemaQuest HQK-1001 Phase 2 study in sickle cell disease

HemaQuest Pharmaceuticals, Inc., a biotechnology company focused on developing small molecule therapeutics to treat hemoglobin disorders, announced today that it has completed enrollment in a randomized multi-dose Phase 2 study of HQK-1001 in patients with sickle cell disease.

12 Sep 2011

Researchers discover new genetic defect that predisposes people to leukemia, myelodysplasia

A new genetic defect that predisposes people to acute myeloid leukemia and myelodysplasia has been discovered. The mutations were found in the GATA2 gene. Among its several regulatory roles, the gene acts as a master control during the transition of primitive blood-forming cells into white blood cells.

5 Sep 2011