Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, announced today that it has presented key pre-clinical proof-of-concept data from its RNAi therapeutic program targeting aminolevulinate synthase-1 (ALAS-1) for the treatment of porphyria including acute intermittent porphyria.
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BGI Health and ACIBADEM Healthcare Group Genetic Diagnostic Center in cooperation with Genoks Company Molecular Biology, signed a Memorandum of Understanding for jointly introducing the advanced genetic testing technologies to Turkey and improving Turkish reproductive healthcare.
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Two studies led by investigators at Weill Cornell Medical College shed light on the molecular biology of three blood disorders, leading to novel strategies to treat these diseases.
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Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced the initiation of a phase 2 study of its novel, investigational protein therapeutic, ACE-536, to treat patients with beta-thalassemia, a genetic hematologic disorder causing chronic and life-threatening anemia and serious complications affecting the spleen, liver and heart.
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Gamida Cell announced today the successful results of the Phase I/II study of its second pipeline product NiCord, umbilical cord derived stem cells expanded using the company's proprietary NAM technology.
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Sangamo BioSciences, Inc. today reported fourth quarter and full year 2012 financial results and accomplishments.
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The U.S. Food and Drug Administration recently announced the authorization of FerriScan R2-MRI to be marketed as an imaging companion diagnostic device for the safe and effective use of Exjade in patients with non-transfusion-dependent thalassemia.
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Acceleron Pharma, Inc., a biopharmaceutical company developing protein therapeutics for cancer and orphan diseases, today announced the initiation of a phase 2 study of its investigational protein therapeutic, ACE-536, to treat anemia in patients with myelodysplastic syndromes (MDS).
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The U.S. Food and Drug Administration today expanded the approved use of Exjade (deferasirox) to treat patients ages 10 years and older who have chronic iron overload resulting from a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT).
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Penn Medicine will establish the Philadelphia region's first dedicated center for the treatment and research of blood diseases by combining the expertise of physicians who specialize in the care of blood disorder patients of all ages along with basic science and clinical researchers who are working to advance treatments for these illnesses.
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Acetylon Pharmaceuticals Inc., a leader in targeted epigenetic drug discovery and development for enhanced therapeutic outcomes, today announced results from a preclinical study of a selective histone deacetylase (HDAC) 1/2 inhibitor for the treatment of sickle cell disease (SCD) and beta-thalassemia (bT) at the 54th Annual Meeting of the American Society of Hematology (ASH), taking place in Atlanta, Georgia.
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Emmaus Medical, Inc., a specialty pharmaceutical and regenerative medicine company, today announced the completion of patient enrollment for its phase 3 clinical trial to study L-glutamine as a treatment for sickle cell disease.
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Novartis will highlight more than 140 presentations on key data from its extensive oncology portfolio at the leading year-end scientific meetings devoted to hematology and breast cancer, demonstrating continued innovation in research and development efforts to advance the care of patients with cancer and rare diseases.
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Acetylon Pharmaceuticals Inc., a leader in targeted epigenetic drug discovery and development for enhanced therapeutic outcomes, today announced that its selective histone deacetylase (HDAC) inhibitors will be featured in five posters and one oral presentation at the 54th Annual Meeting of the American Society of Hematology (ASH), taking place December 8-11, 2012, in Atlanta, Georgia.
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Sickle cell disease is a devastating hereditary condition that affects millions of people, particularly those in West and Central Africa as well as people of African descent in the United States, Europe and South America.
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Bone marrow transplant (also called stem cell transplant) is a medical intervention that allows a physician to deliver very high doses of chemotherapy (if needed) but more importantly, deliver a new immune system to the patient to fight a disease (such as cancer).
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Gamida Cell, a leader in adult stem cell expansion technologies and products, announced today that it has completed enrollment for a Phase I/II clinical trial of NiCord, the company's second pipeline product.
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Sangamo BioSciences, Inc. announced that data from its Phase 1 clinical programs to develop SB-728-T, a novel therapeutic approach designed to generate a "functional cure" for HIV/AIDS, were presented at the 52nd Interscience Conference on Antimicrobial Agents and Chemotherapy (ICAAC).
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bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, today announced the successful completion of a $60 million Series D financing. In this round, new investors Deerfield Partners, RA Capital, Ramius Capital Group, and two undisclosed blue chip public investment funds joined existing investors ARCH Venture Partners, Third Rock Ventures, TVM Capital, and Forbion Capital Partners. In addition, Shire plc joined the round as a strategic investor.
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Vibration therapy may be an effective nonpharmacologic intervention to increase bone mass in patients with thalassemia, US researchers report
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