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.
Key opinions leaders to discuss role of cyclophosphamide in the field of HSCT

Key opinions leaders to discuss role of cyclophosphamide in the field of HSCT

Key opinions leaders in the field of haematopoetic stem cell transplantation (HSCT) will address the role of Cyclophosphamide, an anti-cancer chemotherapy drug, during the 42nd Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) that will welcome more than 4,500 delegates in the host city of Valencia, Spain from the 3rd to the 6th of April 2016. [More]
Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 2016

Sangamo presents immunological data from SB-728-T HIV clinical study at CROI 2016

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced the presentation of immunological data from the Company's clinical trials of SB-728-T, a ZFP Therapeutic designed to provide functional control of HIV. [More]
FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration has cleared the Company's Investigational New Drug (IND) application for SB-318, a single treatment strategy intended to provide a life-long therapy for Mucopolysaccharidosis Type I (MPS I). [More]
Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo presents Phase 2 data from two ongoing clinical trials of SB-728-T for treatment of HIV/AIDS

Sangamo BioSciences, Inc., the leader in therapeutic genome editing, announced the presentation of Phase 2 data from two of the Company's ongoing clinical trials (SB-728-1101 Cohort 3* and SB-728-mR-1401) of SB-728-T, which is being developed for the functional control of HIV/AIDS. [More]
Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

Experimental gene therapy may improve health outcomes for patients with some forms of blood disorders

New research adds to a growing body of evidence that gene therapy, an experimental technique that involves correcting or replacing a person's mutated or malfunctioning genes, may improve health outcomes for patients with inherited bleeding and immune disorders as well as some forms of blood cancer. [More]
Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Boston Children's Hospital selects Vijay G. Sankaran to receive Rising Star Award

Saluting his spectacular track record to date of innovative research on red blood cell disorders and his future promise as a physician/scientist, Boston Children's Hospital has selected Vijay G. Sankaran, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center to receive the Rising Star Award at the hospital's third annual Global Pediatric Innovation Summit, Taking on Tomorrow (#PedInno15). [More]
New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

New method may extend use of noninvasive prenatal testing to detect chromosomal abnormalities

Researchers at the University of California, San Diego School of Medicine developed a method to expand the types of chromosomal abnormalities that noninvasive prenatal testing (NIPT) can detect. The study, published November 9 by Proceedings of the National Academy of Sciences, uses a semiconductor sequencing platform to identify small chromosomal deletions or duplications, such as occur in Cri du Chat Syndrome and DiGeorge Syndrome, with a simple blood test from the expectant mother. [More]
Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Shire and Sangamo revise agreement to expedite development of ZFP Therapeutics for Huntington's disease, hemophilia A and B

Sangamo BioSciences, Inc., a leader in therapeutic genome editing, announced today that the company and its collaborator, Shire plc, have agreed to revise their January 2012 collaboration and license agreement to expedite the development of ZFP Therapeutics for hemophilia A and B and Huntington's disease. [More]
Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Gamida Cell reaches agreements with FDA and EMA regarding NiCord Phase III study design outline

Gamida Cell, a leader in cellular and immune therapies for the treatment of cancer and orphan genetic diseases, announced today that the company has reached agreements with the FDA and EMA regarding the Phase III study design outline of NiCord. [More]
Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

Americord Registry, Smart Cells International partner to educate parents on benefits of cord blood banking

Today, Americord Registry announced a new partnership with Smart Cells International, the first cord blood company in the United Kingdom, as part of its initiatives during Cord Blood Awareness Month (July) to educate parents around the globe on the benefits of cord blood banking. [More]
Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences to present data on ZFP Therapeutic platform at ASGCT meeting

Sangamo BioSciences, Inc. announced that data from clinical, preclinical and research-stage programs focused on the development of ZFP Therapeutics will be presented at the 18th Annual Meeting of the American Society of Gene and Cell Therapy [More]
Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals' SANGUINATE granted FDA Orphan Drug Designation for SCD treatment

Prolong Pharmaceuticals, LLC, a biopharmaceutical company dedicated to developing products for the treatment of anemias, cancers and their debilitating comorbidities, announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its flagship product SANGUINATE for the treatment of Sickle Cell Disease (SCD). [More]
New resource available to help older Chinese Americans better understand their healthcare needs

New resource available to help older Chinese Americans better understand their healthcare needs

On the eve of National Minority Health Month, which helps raise awareness for disparities in health and care among minorities in the U.S., a new resource is available to help one such group, older Chinese Americans, better understand and drive their own well-being. [More]
Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for sickle cell disease

Emmaus Life Sciences presents Phase 3 clinical trial of PGLG treatment for 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 data from the Company’s Phase 3 clinical trial of its pharmaceutical grade L-glutamine (PGLG) treatment for sickle cell anemia and sickle beta-0 thalassemia, will be presented, during the 9th Annual Sickle Cell Disease Research and Educational Symposium and 38th National Sickle Cell Disease Scientific Meeting. [More]
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]
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