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.
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]
Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam presents new pre-clinical data with ALN-AT3 for treatment of hemophilia and RBD

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented new pre-clinical data with ALN-AT3, a subcutaneously administered RNAi therapeutic targeting antithrombin (AT) for the treatment of hemophilia and rare bleeding disorders (RBD), at the 55th Annual Meeting of the American Society of Hematology (ASH) held December 7 - 10, 2013 in New Orleans. [More]
Acetylon announces positive preclinical data of ricolinostat (ACY-1215) in models of lymphoma

Acetylon announces positive preclinical data of ricolinostat (ACY-1215) in models of lymphoma

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase (HDAC) inhibitors for enhanced therapeutic outcomes, announced that positive preclinical data of selective HDAC6 inhibitor, ricolinostat (ACY-1215), in models of lymphoma were presented today in an oral presentation by Dr. Jennifer Amengual, Assistant Professor of Medicine & Experimental Therapeutics, Columbia University Medical Center. [More]
Acetylon Pharmaceuticals presents positive interim data from two ricolinostat clinical trials at ASH 2013

Acetylon Pharmaceuticals presents positive interim data from two ricolinostat clinical trials at ASH 2013

Acetylon Pharmaceuticals, Inc., the leader in the development of selective histone deacetylase inhibitors for enhanced therapeutic outcomes, today announced that positive interim clinical data from the two proof-of-concept clinical trials with selective HDAC6 inhibitor, ricolinostat (ACY-1215), were presented at the 55th Annual Meeting of the American Society of Hematology in New Orleans, LA. [More]
Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

Genetically engineered cell therapies demonstrate early efficacy, safety in patients with blood disorders

A series of advancements in genetically engineered cell therapies demonstrate early efficacy and safety in patients with blood disorders for whom standard treatments have been unsuccessful, according to data showcased today during the 55th American Society of Hematology (ASH) Annual Meeting and Exposition in New Orleans. [More]
Novartis to present updates on broad cancer portfolio at ASH and SABCS symposiums

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). [More]
CHEO-led study confirms validity, reliability of quality of life measurement tool for thalassemia major

CHEO-led study confirms validity, reliability of quality of life measurement tool for thalassemia major

A CHEO-led multi-site North American study, headed by Dr. Robert Klaassen, lead investigator at CHEO and associate professor in the University of Ottawa's Department of Pediatrics, has confirmed the validity and reliability of a quality of life measurement tool for children and adults with thalassemia major. [More]
IVF and chromosome testing: an interview with Dr. Santiago Munné

IVF and chromosome testing: an interview with Dr. Santiago Munné

The Society for Assisted Reproductive Technology (SART) releases every year a report on pregnancy rates by maternal age. The last one shows a decline on implantation rates per embryo from 36% in young women, to 27.3% in women 35-37, 17.5% in 38-40, and less than 10% above 40 years of age. [More]
Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam advances Development Candidate for ALN-AS1 for treatment of hepatic porphyrias

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that it is advancing its Development Candidate for ALN-AS1, an RNAi therapeutic targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP). [More]