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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

Study reveals positive impact of Soliris on long-term outcomes in PNH patients

Alexion Pharmaceuticals, Inc. today announced the presentation of research that provides further insight into the clinical consequences of paroxysmal nocturnal hemoglobinuria (PNH), and positive impact of Soliris® (eculizumab) therapy on long-term outcomes.

13 Jun 2011

Final data from Alexion's Soliris Phase 2 studies on aHUS presented at EHA 2011

Alexion Pharmaceuticals, Inc. today announced the presentation of final data from the two Phase 2 studies of Soliris as a treatment for patients with atypical hemolytic uremic syndrome.

13 Jun 2011

UAB first in U.S. with cell-processing workstation

The University of Alabama at Birmingham has taken a significant step toward making sophisticated cell therapy a part of patient care with its acquisition of the first cell-processing workstation (CPWS) from SANYO North America Corporation (SANYO) in the United States.

8 Jun 2011

Alexion's sBLA for Soliris receives FDA Priority Review designation to treat aHUS

Alexion Pharmaceuticals, Inc. announced today that the U.S. Food and Drug Administration has granted the Company's request for Priority Review of its supplemental Biologics License Application for Soliris as a treatment for patients with atypical Hemolytic Uremic Syndrome.

1 Jun 2011

Birth control pill safety issues raised

The U.S. Food and Drug Administration (FDA) has announced that it is looking at results from two studies that showed there is a higher risk of blood clots in women taking new birth control pills containing the compound drospirenone. Some brand names include Yaz, Yasmin, Beyaz, Ocella, Loryna, Gianvi, Safyral, Syeda and Zarah.

1 Jun 2011

CSL-Behring recombinant factor research presented

Advances that CSL Behring is making in developing innovative new technologies to meet the unmet therapeutic needs of patients with hemophilia A and B will be the topic of an oral presentation at an international medical congress on May 22, 2011. CSL Behring is a global biotherapeutics company specializing in plasma-derived and recombinant therapies and is a subsidiary of CSL Limited (ASX:CSL).

19 May 2011

Doctors miss blood clots costing millions to the NHS: Report

According to to figures from the NHS Litigation Authority, the NHS has paid out over £112m since 2005 in compensation because doctors have failed to spot or prevent fatal blood clots.

12 May 2011

$Hyponatremia linked to high risk of fractures in elderly patients$

Hyponatremia linked to high risk of fractures in elderly patients

A common blood disorder, hyponatremia, may be a risk factor for fractures in elderly patients, new research presented at the European Congress of Endocrinology shows.

3 May 2011

HemaQuest initiates 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 clinical investigators have enrolled the first patient in a randomized multi-dose Phase 2 study of HQK-1001 in patients with sickle cell disease.

26 Apr 2011