Thalassemia News and Research RSS Feed - Thalassemia News and Research

Thalassemia is a group of genetic blood disorders that affect approximately 1,000 individuals in the United States. The most severe of these disorders is Cooley's Anemia.

People with thalassemia have a genetic defect of their red blood cells that affects the cells' ability to produce normal hemoglobin. Red blood cells use hemoglobin to carry oxygen to tissues. As a result of the defect, most forms of thalassemia produce a chronic, lifelong anemia that begins in early childhood and often must be treated with frequent transfusions.
MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

MRI more accurate, safe and less painful than liver biopsy in measuring total body iron balance

Investigators at The Saban Research Institute of Children's Hospital Los Angeles have demonstrated that MR imaging of the liver is more accurate than liver biopsy in determining total body iron balance in patients with sickle cell disease and other disorders requiring blood transfusion therapy. [More]
New bone marrow transplant unit launched in Bangalore, India

New bone marrow transplant unit launched in Bangalore, India

A bone marrow transplant can mean the difference between life and death for people with blood cancers and related disorders. But many patients in India can't afford the high treatment costs, and for them a transplant is not an option. This is changing thanks to a newly launched bone marrow transplant unit at M.S. Ramaiah Medical College in Bangalore. [More]
FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

FDA accepts Sangamo BioSciences' IND for SB-BCLmR-HSPC genome editing approach

Sangamo BioSciences, Inc. announced today that an Investigational New Drug (IND) application for the company's SB-BCLmR-HSPC genome editing approach, which is designed to provide a one-time lasting therapy for beta-thalassemia, has been accepted by the U.S. Food and Drug Administration and is now active. [More]
Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus to exhibit new mRNA therapeutics pipeline at two healthcare conferences

Arcturus Therapeutics Inc., developing the 'next wave' of RNA medicines, today announced that Joseph Payne, President and Chief Executive Officer, is scheduled to present a corporate overview, including the company's new messenger RNA (mRNA) therapeutics pipeline, at two upcoming healthcare conferences. [More]
Gamida Cell granted FDA and EMA orphan drug designation for NiCord

Gamida Cell granted FDA and EMA orphan drug designation for NiCord

Gamida Cell, a leader in cell therapy technologies and products for transplantation and adaptive immune therapy, announced today that orphan drug designation has been granted by The US Department of Health and Human Services, The FDA Office of Orphan Products Development (OOPD) for the investigational medicinal product NiCord for the treatment of acute lymphoblastic leukemia (ALL), acute myeloid leukemia (AML), Hodgkin lymphoma and myelodysplastic syndrome (MDS). [More]
Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Emerging strategies to boost healthy RBCs may reduce burden of anemia linked to blood disorders

Emerging treatment approaches may reduce the burden of anemia associated with blood disorders by enhancing production of healthy red blood cells, according to data presented today at the 56th American Society of Hematology Annual Meeting and Exposition. [More]
Genom Austria project to explore impact of genome sequencing on science and society

Genom Austria project to explore impact of genome sequencing on science and society

In many countries, genome sequencing technology is now starting to become available in the clinic, where it helps to diagnose rare Mendelian diseases and contributes to personalized cancer therapy. The analysis of personal genomes also creates unprecedented opportunities for predictive health counseling, ancestry research, and many more applications that are just starting to emerge. [More]
BloodCenter's Erythroid Chimerism test available to monitor transplanted SCD patients

BloodCenter's Erythroid Chimerism test available to monitor transplanted SCD patients

BloodCenter of Wisconsin's Diagnostic Laboratories today announced the availability of an innovative Erythroid Chimerism test to monitor erythroid lineage chimerism in patients with sickle cell disease (SCD) following allogeneic bone marrow transplantation. [More]
Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam announces new pre-clinical results with DC for ALN-CC5 to treat complement-mediated diseases

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today new pre-clinical results with its Development Candidate (DC) for ALN-CC5, a subcutaneously administered investigational RNAi therapeutic targeting complement component C5 in development for the treatment of complement-mediated diseases. [More]
Findings could lead to treatments for blood disorders associated with iron deficiencies and overloads

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. [More]
CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

CIRM grants $5.6 million to develop HIV/AIDS therapeutic using Sangamo's ZFN genome-editing technology

Sangamo BioSciences, Inc. announced that the California Institute for Regenerative Medicine (CIRM) has granted a $5.6 million Strategic Partnership Award to fund clinical studies at City of Hope to develop a potentially curative ZFP Therapeutic for HIV/AIDS based on the application of Sangamo's zinc finger nuclease (ZFN) genome-editing technology in hematopoietic stem/progenitor cells (HSPCs). [More]
Immucor's PreciseType HEA test receives FDA approval to predict blood compatibility

Immucor's PreciseType HEA test receives FDA approval to predict blood compatibility

Immucor, Inc., a global leader in transfusion and transplantation diagnostics, announced that the U.S. Food and Drug Administration has granted approval for the company's PreciseType HEA test. [More]
Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

Acceleron, Celgene to give oral presentations on beta-thalassemia, myelodysplastic syndromes at EHA meeting

Acceleron Pharma Inc., a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of novel protein therapeutics for cancer and rare diseases, today reported that Acceleron, Celgene and investigators in the sotatercept and ACE-536 phase 2 clinical trials will give three oral presentations of interim data from ongoing studies in beta-thalassemia and myelodysplastic syndromes as well as a poster presentation of nonclinical data in sickle cell disease at the 19th Annual Congress of the European Hematology Association (EHA) in Milan, Italy from June 12-15, 2014. [More]
Scientists design new system for molecular blood group typing

Scientists design new system for molecular blood group typing

Scientists in France have designed a new system for molecular blood group typing that offers blood banks the possibility of extensive screening of blood donors at a relatively low cost. Their approach is described in the current issue of The Journal of Molecular Diagnostics. [More]
Emmaus reports preliminary top-line results from Phase 3 clinical trial for treatment of sickle cell disease

Emmaus reports preliminary top-line results from Phase 3 clinical trial for treatment of sickle cell disease

Emmaus Life Sciences, Inc., a biopharmaceutical company dedicated primarily to the discovery, development and commercialization of innovative treatments and therapies for rare and orphan diseases, today announced that preliminary top-line results of its Phase 3 clinical trial evaluating the safety and efficacy of its treatment for sickle cell anemia and sickle beta-0 thalassemia met both the primary and secondary endpoints of the clinical trial. [More]
Biogen Idec, Sangamo BioSciences partner to develop therapeutics for hemoglobinopathies

Biogen Idec, Sangamo BioSciences partner to develop therapeutics for hemoglobinopathies

Biogen Idec and Sangamo BioSciences, Inc. announced today an exclusive worldwide collaboration and license agreement focused on the development of therapeutics for hemoglobinopathies, inherited conditions that result from the abnormal structure or underproduction of hemoglobin. [More]

Emmaus completes all patient visits for Phase 3 trial of L-glutamine for treatment of sickle cell disease

Emmaus Medical, Inc., a biopharmaceutical company engaged in the discovery, development and commercialization of innovative and cost-effective treatments and therapies primarily for rare and orphan diseases, today announced it has completed all patient visits for its Phase 3 clinical trial for the treatment of sickle cell disease. Emmaus expects top-line data from the Phase 3 clinical trial to be available in the first quarter of 2014. [More]
New projects advance treatments for radiation syndrome, cardiac arrest-induced brain injury and rare blood disorder

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. [More]
Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam initiates Phase II study with ALN-TTRsc for treatment of ATTR

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has initiated a pilot Phase II study with ALN-TTRsc, a subcutaneously delivered RNAi therapeutic targeting the transthyretin gene in development for the treatment of TTR-mediated amyloidosis. [More]
Acetylon Pharmaceuticals presents positive preclinical data for selective HDAC1/2 inhibitors at ASH meeting

Acetylon Pharmaceuticals presents positive preclinical data for selective HDAC1/2 inhibitors at ASH meeting

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, announced that positive preclinical data for its selective HDAC1/2 inhibitors for the treatment of sickle cell disease (SCD) and β-thalassemia (bT) were presented at the 55th Annual Meeting of the American Society of Hematology (ASH) in New Orleans, LA. [More]
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