Thalassemia News and Research

Latest Thalassemia News and Research

Medication and blood transfusion reduce risk of recurrent strokes in young patients with sickle cell anemia

St. Jude Children's Research Hospital investigators discuss results of research into managing iron overload associated with stroke prevention in young sickle cell patients and the search for genetic predictors of stroke risk.

7 Dec 2010

Research highlighting treatment for pediatric SCD, malaria presented at ASH 2010

Red blood cells have the very important role of carrying oxygen from the lungs to the rest of the body; therefore, disorders that affect RBCs can have a significant impact on quality of life. Sickle cell disease and malaria are two common RBC diseases that affect a significant portion of the U.S. and global populations. Sickle cell disease, a genetic blood disorder that causes deformed and dysfunctional red blood cells, affects an estimated 70,000 to 100,000 Americans, while malaria affects approximately 1,500 Americans annually and 190 to 311 million people worldwide.

6 Dec 2010

Study compares efficacy of two treatments for iron overload caused by chronic transfusion therapy

Monthly blood transfusions combined with daily medication to remove the resulting excess iron remains the best approach for reducing the risk of recurrent strokes in young patients with sickle cell anemia, according to a preliminary analysis of a multicenter trial that includes St. Jude Children's Research Hospital.

6 Dec 2010

Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

A hormone made by the body may be a potential therapeutic tool for the treatment of two anemic blood disorders beta-thalassemia and hemochromatosis. The new research was led by scientists at Weill Cornell Medical College and published in the Journal of Clinical Investigation and the journal Blood.

24 Nov 2010

ENERCA network organizes European Symposium on rare anemias

It is estimated that rare diseases -those whose frequency is under 5 cases / 10,000 people- affect about 6% of the European population. In the field of anemia, 1% of couples are at risk of having a newborn with a severe syndrome of hemoglobin. More than 330,000 children are born worldwide each year affected by one of these diseases, being the most common disorders sickle cell anemia and thalassemia syndromes.

19 Nov 2010

CINJ experts to comment on importance of blood donation

Every two seconds, there is someone in the United States in need of blood. According to the American Red Cross, 65 percent of the American population is eligible to donate blood, but only five percent actually do. In New Jersey only two and a half percent of eligible donors will donate, while nine out of ten New Jersey residents will need blood at least once in their lives.

19 Nov 2010

New mathematical model may help predict anemia risk

A collaboration between a physician-researcher at Massachusetts General Hospital (MGH) and a mathematician from Harvard University has led to development of a mathematical model reflecting how red blood cells change in size and hemoglobin content during their four-month lifespan.

15 Nov 2010

StemCyte receives two cash grants under QTDP program for biomedical research

StemCyte, Inc., one of the world's preeminent umbilical cord blood (UCB) banks and biotherapy companies, is proud to announce that the Company has been awarded two cash grants totaling $488,950 under the U.S. Internal Revenue Service's Qualifying Therapeutic Discovery Project (QTDP) program designed to support biomedical research by firms with fewer than 250 employees.

11 Nov 2010

Merck's boceprevir Phase III studies for HCV to be presented at 61st AASLD

Merck today announced that final results from two pivotal Phase III studies of boceprevir, its investigational oral hepatitis C protease inhibitor, will be presented in oral plenary sessions at the 61st Annual Meeting of the American Association for the Study of Liver Diseases (AASLD), which is taking place from Oct. 29 through Nov. 2 in Boston. Results for boceprevir in response-guided therapy strategies, which evaluated treatment durations shorter than current standard therapy, will be presented during the meeting.

4 Oct 2010

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

bluebird bio an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced publication in the journal Nature of its promising Phase 1/2 data highlighting positive results of LentiGlobin gene therapy treatment in a young adult with severe beta-thalassemia, a blood disorder that is one of the most frequent inherited diseases.

16 Sep 2010

FerroKin BioSciences initiates international Phase 2 study of FBS0701

FerroKin BioSciences today announced the initiation of an international Phase 2 study of FBS0701, a novel once-daily iron chelator in development for the treatment of transfusional iron overload. The Phase 2 study will assess the safety, tolerability, and pharmacodynamics of FBS0701.

10 Sep 2010

HemaQuest commences HQK-1004 global Phase 2 clinical trial in EBV-related malignancies

HemaQuest Pharmaceuticals announced today that it has initiated the Company's Phase 2 clinical trial evaluating its proprietary HQK-1004 therapy for the treatment of lymphomas and related cancers associated with Epstein-Barr virus (EBV) infection. These EBV-related cancers have poor prognoses and short patient survival. Currently available cancer therapies are typically much less effective in patients with EBV-related lymphoid malignancies than in patients who have similar cancers not infected with the virus.

11 Aug 2010

Merck's two pivotal boceprevir Phase III registration studies for chronic HCV meet primary endpoint

Merck reported that two pivotal Phase III registration studies for boceprevir, its investigational oral hepatitis C protease inhibitor, have been completed and met the primary endpoints: in both studies in patients with chronic hepatitis C virus (HCV) genotype 1 infection, the addition of boceprevir to treatment with PEGINTRON® and REBETOL® significantly increased the number of patients who achieved sustained virologic response, compared to control groups that received Peg/riba plus placebo.

5 Aug 2010

HemaQuest closes additional $4M to Series B financing

HemaQuest Pharmaceuticals, a clinical stage biotechnology company developing small molecule therapeutics for sickle cell disease, beta thalassemia and EBV-related cancers, announced today the closing of an additional $4M to its Series B financing by new investor, Latterell Venture Partners. This investment brings the total Series B financing to $16M.

28 Jul 2010

Merck signs non-exclusive license agreement with LabCorp for commercialization of genetic test

Merck announced a non-exclusive license agreement with Laboratory Corporation of America Holdings for the commercialization of a genetic test that may help predict the response of patients with Hepatitis C virus infection to peginterferon alpha-based therapy.

28 Jul 2010

Experimental treatment for sickle cell disease enters Phase 3 clinical trials

An experimental treatment for sickle cell disease developed at the Los Angeles Biomedical Research Institute has entered Phase 3 clinical trials, David I. Meyer, PhD, LA BioMed president and CEO announced today.

26 Jul 2010

Researchers discover genetic explanation for non-diabetic kidney disease in African-Americans

Kidney disease is a growing public health problem, with approximately half a million individuals in the United States requiring dialysis treatments to replace the function of their failed kidneys. The problem is particularly acute among African-Americans, whose rates of kidney disease are four times higher than those of European Americans.

20 Jul 2010

Gene therapy represents safe alternative to current cures for blood disorder β-thalassemia

Italian scientists pioneering a new gene transfer treatment for the blood disorder β-thalassemia have successfully completed preclinical trials, claiming they can correct the lack of beta-globin (β-globin) in patients' blood cells which causes the disease. The research, published in EMBO Molecular Medicine, reveals how gene therapy may represent a safe alternative to current cures that are limited to a minority of patients.

14 Jul 2010

CorMedix announces dosing of first patient with CRMD-001 in Phase II clinical trial

CorMedix Inc., a pharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of Cardiorenal disease, today announced dosing of the first patient with CRMD-001 (a proprietary formulation of deferiprone) in a randomized, double-blind, placebo-controlled clinical trial.

25 Jun 2010

NHLBI funds nine research grants to determine safety and efficacy of red blood cell transfusions

The National Heart, Lung, and Blood Institute, part of the National Institutes of Health, is funding nine research grants to determine if the safety and efficacy of red blood cell transfusions vary depending on how long the cells have been stored. One of the grants supports the first large, multi-center, randomized clinical trial to compare outcomes in heart surgery patients who receive transfusions of red blood cells that have been stored for shorter or longer amounts of time.

22 Jun 2010

Thalassemia News and Research

Latest Thalassemia News and Research

Medication and blood transfusion reduce risk of recurrent strokes in young patients with sickle cell anemia

Research highlighting treatment for pediatric SCD, malaria presented at ASH 2010

Study compares efficacy of two treatments for iron overload caused by chronic transfusion therapy

Hepcidin hormone may be potential therapeutic tool for anemic blood disorders

ENERCA network organizes European Symposium on rare anemias

CINJ experts to comment on importance of blood donation

New mathematical model may help predict anemia risk

StemCyte receives two cash grants under QTDP program for biomedical research

Merck's boceprevir Phase III studies for HCV to be presented at 61st AASLD

bluebird bio publishes LentiGlobin gene therapy clinical trial data results

FerroKin BioSciences initiates international Phase 2 study of FBS0701

HemaQuest commences HQK-1004 global Phase 2 clinical trial in EBV-related malignancies

Merck's two pivotal boceprevir Phase III registration studies for chronic HCV meet primary endpoint

HemaQuest closes additional $4M to Series B financing

Merck signs non-exclusive license agreement with LabCorp for commercialization of genetic test

Experimental treatment for sickle cell disease enters Phase 3 clinical trials

Researchers discover genetic explanation for non-diabetic kidney disease in African-Americans

Gene therapy represents safe alternative to current cures for blood disorder β-thalassemia

CorMedix announces dosing of first patient with CRMD-001 in Phase II clinical trial

NHLBI funds nine research grants to determine safety and efficacy of red blood cell transfusions

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Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

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Thalassemia News and Research

Latest Thalassemia News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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