Blood Disorder News and Research

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Penn Medicine-developed drug gets FDA orphan status for PNH treatment

A Penn Medicine-developed drug has received orphan status from the Food and Drug Administration (FDA) this month for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.

28 Oct 2014

Molecular diagnostics and blood screening systems: an interview with Dr Bos and Dr Koppelman

Screening blood donations and molecular diagnostics for patients started in the nineties. At the beginning, the level of automation in molecular diagnostic testing was limited; most of the work had to be done manually.

12 Oct 2014

Research performed in anemic mice shows acetate stimulates the formation of red blood cells

UT Southwestern Medical Center researchers seeking novel treatments for anemia found that giving acetate, the major component of household vinegar, to anemic mice stimulated the formation of new red blood cells.

30 Sep 2014

Scientists identify gene mutation that causes aplastic anemia

An international team of scientists has identified a gene mutation that causes aplastic anemia, a serious blood disorder in which the bone marrow fails to produce normal amounts of blood cells.

23 Sep 2014

Simple point-of-care testing device provides more rapid diagnosis of anemia

A simple point-of-care testing device for anemia could provide more rapid diagnosis of the common blood disorder and allow inexpensive at-home self-monitoring of persons with chronic forms of the disease.

15 Sep 2014

Penn receives orphan status in Europe for treatment of PNH

A Penn Medicine-developed drug has received orphan status in Europe this week for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease that causes anemia due to destruction of red blood cells and thrombosis.

27 Aug 2014

FDA-approved drug eliminates immune cells that destroys hair follicles in people with alopecia areata

Researchers at Columbia University Medical Center (CUMC) have identified the immune cells responsible for destroying hair follicles in people with alopecia areata, a common autoimmune disease that causes hair loss, and have tested an FDA-approved drug that eliminated these immune cells and restored hair growth in a small number of patients.

18 Aug 2014

Baxter International acquires drug candidate developed to treat sickle cell disease

A drug candidate developed by researchers at the NIH's National Center for Advancing Translational Sciences and its collaborators to treat sickle cell disease has been acquired by Baxter International's BioScience business.

11 Jul 2014

Research explores links between planet's health and human health

United Nations University will help pioneer a fresh trail in global health research, exploring links between the planet's health and human health at an institute in Kuala Lumpur generously supported by Malaysia.

17 Jun 2014

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Soliris® (eculizumab) for the treatment of patients with Myasthenia Gravis (MG), a rare, debilitating neurologic disorder caused by uncontrolled complement activation.

17 Jun 2014

Provepharm, DFKC partner to launch Methylthioninium chloride Proveblue in Germany

Provepharm, a French company specialized in the development of pharmaceutical applications, and Dr Franz Kohler Chemie, a German leader in the manufacture and supply of organ preservation solutions and antidote pharmaceuticals, today announce that they have entered into a partnership to launch Provepharm’s drug product Methylthioninium chloride Proveblue 5mg/ml solution for injection (the Product) in Germany, for the acute symptomatic treatment of medicinal and chemical products-induced methaemoglobinaemia.

17 Jun 2014

Findings could lead to treatments for blood disorders associated with iron deficiencies and overloads

A UCLA research team has discovered a new hormone called erythroferrone, which regulates the iron supply needed for red blood-cell production.

2 Jun 2014

FDA approves Alexion’s Soliris drug for treatment of atypical hemolytic uremic syndrome

Alexion Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has approved the Company's supplemental Biologics License Application (sBLA) providing regular approval for Soliris® (eculizumab) for the treatment of adult and pediatric patients with atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA).

6 May 2014

Alexion Pharmaceuticals obtains orphan drug designation from EC for Soliris

Alexion Pharmaceuticals, Inc. today announced that the European Commission has granted an orphan drug designation (ODD) to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of graft rejection following solid organ transplantation. Graft rejection can cause severe injury to the transplanted organ and is a significant barrier to successful transplantation.

23 Apr 2014

Penn research team discovers new cost-effective way to fight rare blood disorder

A University of Pennsylvania research team has defined a possible new way to fight a disease that is currently treatable only with the most expensive drug available for sale in the United States.

28 Feb 2014

Penn researchers identify possible new way to fight rare blood disorder

A University of Pennsylvania research team has defined a possible new way to fight a disease that is currently treatable only with the most expensive drug available for sale in the United States. In a study published this month in Blood, the Penn team describes the strategy, based on the oldest part of the human immune system - called "complement" -- that could turn out to be less costly and more effective for the majority of patients with a rare blood disorder.

27 Feb 2014

Blood disorder medicines could be used for treating stomach and bowel cancer

Stomach and bowel cancer, two of the most common cancers worldwide, could be treated with a class of medicines that are currently used to treat a blood disorder, a Melbourne research team has discovered.

5 Feb 2014

Alexion Pharmaceuticals reports full year 2013 GAAP and non-GAAP financial results

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter and year ended December 31, 2013. For the three months ended December 31, 2013, Alexion Pharmaceuticals, Inc. ("Alexion" or the "Company") reported net product sales of Soliris® (eculizumab) of $441.9 million, compared to $320.5 million for the same period in 2012.

30 Jan 2014

Alexion's Soliris gets FDA orphan drug designation for DGF prevention in renal transplant patients

Alexion Pharmaceuticals today announced that the U.S. Food and Drug Administration has granted an orphan drug designation to Soliris (eculizumab), a first-in-class terminal complement inhibitor, for the prevention of delayed graft function in renal transplant patients.

21 Jan 2014

New projects advance treatments for radiation syndrome, cardiac arrest-induced brain injury and rare blood disorder

The National Institutes of Health today has launched three pre-clinical projects to advance potential new treatments for acute radiation syndrome, brain injury following cardiac arrest and a rare blood disorder called beta thalassemia.

17 Dec 2013

Blood Disorder News and Research

Penn Medicine-developed drug gets FDA orphan status for PNH treatment

Molecular diagnostics and blood screening systems: an interview with Dr Bos and Dr Koppelman

Research performed in anemic mice shows acetate stimulates the formation of red blood cells

Scientists identify gene mutation that causes aplastic anemia

Simple point-of-care testing device provides more rapid diagnosis of anemia

Penn receives orphan status in Europe for treatment of PNH

FDA-approved drug eliminates immune cells that destroys hair follicles in people with alopecia areata

Baxter International acquires drug candidate developed to treat sickle cell disease

Research explores links between planet's health and human health

Alexion announces that FDA grants ODD to Soliris for Myasthenia Gravis treatment

Provepharm, DFKC partner to launch Methylthioninium chloride Proveblue in Germany

Findings could lead to treatments for blood disorders associated with iron deficiencies and overloads

FDA approves Alexion’s Soliris drug for treatment of atypical hemolytic uremic syndrome

Alexion Pharmaceuticals obtains orphan drug designation from EC for Soliris

Penn research team discovers new cost-effective way to fight rare blood disorder

Penn researchers identify possible new way to fight rare blood disorder

Blood disorder medicines could be used for treating stomach and bowel cancer

Alexion Pharmaceuticals reports full year 2013 GAAP and non-GAAP financial results

Alexion's Soliris gets FDA orphan drug designation for DGF prevention in renal transplant patients

New projects advance treatments for radiation syndrome, cardiac arrest-induced brain injury and rare blood disorder

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