Thalassemia News and Research

Latest Thalassemia News and Research

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

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

Acceleron commences ACE-536 Phase 1 clinical trial in anemia

Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases today announced that it has initiated a Phase 1 clinical study of ACE-536.

14 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

FDA clears Resonance Health's MRI-Q System for cardiac T2* iron assessment

Resonance Health Ltd, a medical device company specializing in imaging core laboratory services has received FDA clearance to market its MRI-Q System.

16 Aug 2011

EGT transfers clinical grade lentiviral vector to MSKCC

Errant Gene Therapeutics, LLC, a pioneering boutique drug development firm specializing in Rare Diseases, announced the transfer of its clinical grade lentiviral vector, TNS 9.55.3, to Memorial Sloan Kettering Cancer Center.

29 Jul 2011

CorMedix completes enrollment in CRMD001 Phase II trial for treatment of acute kidney injury

CorMedix Inc., a pharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of cardiorenal disease, announced it successfully completed patient enrollment in its phase II Contrast-Induced Acute Kidney Injury study with its oral drug candidate CRMD001.

14 Jun 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

bluebird bio announces receipt of additional $30 million in financing

bluebird bio, an emerging leader in the development of innovative gene therapies for severe genetic disorders, today announced that the company has secured an additional $30 million in financing.

20 Apr 2011

CorMedix reports encouraging interim results from CRMD001 phase II study in Contrast Induced Acute Kidney Injury

CorMedix Inc., a pharmaceutical company focused on developing and commercializing therapeutic products for the prevention and treatment of cardiorenal disease, today announced its interim analysis and clinical update for CRMD001.

1 Apr 2011

Merck's VICTRELIS phase III study results against HCV presented at EASL annual meeting

Merck, known as MSD outside of the United States and Canada, announced results from several new data analyses from the pivotal Phase III studies evaluating the addition of its investigational oral protease inhibitor VICTRELIS to peginterferon alfa-2b and ribavirin in adult patients with chronic hepatitis C virus genotype 1 infection.

1 Apr 2011

Merck to present VICTRELIS phase III study data against HCV at EASL annual meeting

Merck, known as MSD outside of the United States and Canada, announced that several new data analyses from Phase III studies of VICTRELIS, its investigational oral hepatitis C protease inhibitor, will be presented at The International Liver CongressTM / 46th European Association for the Study of the Liver annual meeting.

9 Mar 2011

Metallic copper surfaces kill bacteria on contact

Metallic copper surfaces kill microbes on contact, decimating their populations, according to a paper in the February 2011 issue of the journal Applied and Environmental Microbiology. They do so literally in minutes, by causing massive membrane damage after about a minute's exposure, says the study's corresponding author, Gregor Grass of the University of Nebraska, Lincoln. This is the first study to demonstrate this mechanism of bacteriocide.

17 Feb 2011

Researchers report gene therapy strategy that improves Beta Thalassemia in mice model

Researchers at Nationwide Children's Hospital report a gene therapy strategy that improves the condition of a mouse model of an inherited blood disorder, Beta Thalassemia.

11 Jan 2011

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

Thalassemia News and Research

Latest Thalassemia News and Research

ApoPharma receives FDA approval for Ferriprox to treat transfusional iron overload

ODAC recommends ApoPharma's Ferriprox for FDA approval to treat transfusional iron overload

Acceleron commences ACE-536 Phase 1 clinical trial in anemia

Enrollment complete in HemaQuest HQK-1001 Phase 2 study in sickle cell disease

FDA clears Resonance Health's MRI-Q System for cardiac T2* iron assessment

EGT transfers clinical grade lentiviral vector to MSKCC

CorMedix completes enrollment in CRMD001 Phase II trial for treatment of acute kidney injury

HemaQuest initiates HQK-1001 Phase 2 study in sickle cell disease

bluebird bio announces receipt of additional $30 million in financing

CorMedix reports encouraging interim results from CRMD001 phase II study in Contrast Induced Acute Kidney Injury

Merck's VICTRELIS phase III study results against HCV presented at EASL annual meeting

Merck to present VICTRELIS phase III study data against HCV at EASL annual meeting

Metallic copper surfaces kill bacteria on contact

Researchers report gene therapy strategy that improves Beta Thalassemia in mice model

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

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

Thalassemia News and Research

Latest Thalassemia News and Research

Expanding research and clinical options for children with cancer

Examining how pain could play a direct protective role in the gut

From Puberty to Menopause: Clue’s CEO, Audrey Tsang on the Power of Femtech

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