Blood Disorder News and Research

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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

New test to help physicians identify patients with most lethal forms of triple-negative breast cancer

A new test has the potential to help physicians identify patients with the most lethal forms of triple-negative breast cancer, a disease which requires aggressive and innovative treatment.

12 Dec 2013

State highlights: Health care price data and transparency in N.Y. and Calif.; states shift tobacco settlement funds away from prevention

Just how expensive is your hospital? In New York, the answer may lie in a trove of hospital cost data newly posted online by the State Health Department. As part of an effort to make health care pricing more transparent, the state is naming hospitals and listing their median charges and costs for 1,400 conditions and procedures from 2009 to 2011.

11 Dec 2013

First Edition: December 10, 2103

Today's early morning highlights from the major news organizations, including reports about how health law advocates are developing new messaging strategies.

10 Dec 2013

Novartis to present updates on broad cancer portfolio at ASH and SABCS symposiums

Novartis will present updates on its broad cancer portfolio with more than 240 abstracts at the upcoming American Society of Hematology (ASH) annual meeting and CTRC-AACR San Antonio Breast Cancer Symposium (SABCS).

4 Dec 2013

NIH awards 10 new grants to support genomics research in Africa

The National Institutes of Health has awarded 10 new grants totaling up to $17 million over the next four years to support genomics research in Africa, as part of the Human Heredity and Health in Africa (H3Africa) program

2 Oct 2013

Rigel plans to focus on completing three clinical programs for ITP, DLE and dry eye disease

Rigel Pharmaceuticals, Inc. today announced its plans to focus the Company's resources on the completion of three lead clinical programs. These efforts include commencing a Phase 3 clinical study of fostamatinib, an oral SYK inhibitor, in Immune Thrombocytopenic Purpura pending discussions with regulatory agencies.

5 Sep 2013

Drug for children with sickle cell anemia reduces hospitalizations and treatment costs

A drug proven effective for treatment of adults and children with sickle cell anemia reduced hospitalizations and cut annual estimated medical costs by 21 percent for affected infants and toddlers, according to an analysis led by St. Jude Children's Research Hospital. The report appears today in the advance online edition of the journal Pediatrics.

2 Sep 2013

UTHealth researchers find cause for rare type of bleeding disorder

Fifteen years ago, a hematologist came to Dianna Milewicz, M.D., Ph.D., with a puzzle: Multiple generations of an East Texas family suffered from a moderately severe bleeding disorder, but it wasn't hemophilia.

28 Aug 2013

Miniscule signaling molecule plays critical role in certain acute and chronic diseases

Scientists seeking to harness the healing power of a miniscule signaling molecule that plays a critical role in certain acute and chronic diseases have been awarded a Program Project Grant (PPG) of nearly $10 million by the National Heart, Lung and Blood Institute of the National Institutes of Health.

1 Aug 2013

UCSF researchers find a way to knock down cancers caused by tumor-driving protein

UC San Francisco researchers have found a way to knock down cancers caused by a tumor-driving protein called -myc,- paving the way for patients with myc-driven cancers to enroll in clinical trials for experimental treatments.

20 Jul 2013