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.
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 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, 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, 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 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]

Scientists uncover new approach to develop induced pluripotent stem cells for in vivo disease modeling

In a paper in Cell Stem Cell, a team led by researchers in the Boston Children's Hospital's Stem Cell Transplantation Program reports a new approach for turning induced pluripotent stem cells (iPSCs) into hematopoietic stem and progenitor cells for in vivo disease modeling. [More]

Study: Ciclopirox drug completely eradicates infectious HIV from cell cultures

The topical anti-fungal drug Ciclopirox causes HIV-infected cells to commit suicide by jamming up the cells' powerhouse, the mitochondria—according to a study by researchers at Rutgers New Jersey Medical School. And unlike current anti-HIV drugs, Ciclopirox completely eradicates infectious HIV from cell cultures, with no rebound of virus when the drug is stopped. [More]
Global Industry Analysis Report on Companion Diagnostics Market

Global Industry Analysis Report on Companion Diagnostics Market

New Market Research Report Added in MarketResearchReports.Biz Reports Database Companion Diagnostics Market - Global Industry Analysis, Size, Share, Growth, Trends And Forecast, 2013 - 2019 [More]
Sangamo BioSciences signs definitive agreement to buy Ceregene

Sangamo BioSciences signs definitive agreement to buy Ceregene

Sangamo BioSciences, Inc. announced that it signed a definitive agreement to acquire Ceregene, Inc., a privately held biotechnology company focused on developing adeno-associated virus gene therapies. [More]

FDA grants Orphan Drug Designation to Alnylam ALN-AT3 for treatment of hemophilia B

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, announced today that the U.S. Food & Drug Administration (FDA) has granted an Orphan Drug Designation to ALN-AT3 as a therapeutic for the treatment of hemophilia B. [More]