Blood Disorder News and Research

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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

Researchers identify elusive gene responsible for Grey Platelet Syndrome

Researchers have identified an elusive gene responsible for Grey Platelet Syndrome, an extremely rare blood disorder in which only about 50 known cases have been reported. As a result, it is hoped that future cases will be easier to diagnose with a DNA test.

26 Jul 2011

Researchers discover novel combination of two mutations in a patient with ADHD

Often enough, in science as in life, unexpected knowledge has a personal impact. Researchers seeking rare gene variants in just a few individuals with attention-deficit hyperactivity disorder (ADHD) discovered that one patient had a novel combination of two mutations.

21 Jul 2011

Alexion second quarter net product sales of Soliris increase to $185.7 million

Alexion Pharmaceuticals, Inc. today announced financial results for the three and six months ended June 30, 2011. Alexion Pharmaceuticals, Inc. reported net product sales of Soliris of $185.7 million in Q2, reflecting steady addition of new patients, compared to $125.8 million for the same period in 2010.

21 Jul 2011

Young people with serious genetic blood disorder are not getting right help at school

A new study suggests young people with a serious genetic blood disorder are not getting the right help at school, especially pupils who miss lessons due to sickness.

20 Jul 2011

New genetic research holds promise for patients with Chuvash polycythemia

New research conducted at UNC Lineberger Comprehensive Cancer Center, offers hope for people with a rare disorder called Chuvash polycythemia.

23 Jun 2011

Alexion begins eculizumab clinical trial in Shiga-toxin producing E. coli hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. and Alexion Pharma International Sàrl (APIS), announced today that the Paul-Ehrlich-Institut, Germany's healthcare regulatory body for biological products, has authorized initiation of an open-label clinical trial to investigate eculizumab as a treatment for patients with Shiga-toxin producing E. coli hemolytic uremic syndrome, which has resulted from infections by Enterohemorrhagic Escherichia coli in an unusually wide outbreak in Germany that began in May.

20 Jun 2011