Thalassemia News and Research

Latest Thalassemia News and Research

Resonance Health receives FDA marketing authorization for FerriScan R2-MRI device

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.

31 Jan 2013

Acceleron commences ACE-536 phase 2 study in myelodysplastic syndromes

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).

30 Jan 2013

Novartis receives FDA approval for Exjade to treat chronic iron overload in NTDT patients

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).

23 Jan 2013

Penn Medicine to establish new center for treatment and research of blood diseases

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.

18 Jan 2013

Acetylon announces results from selective HDAC 1/2 inhibitor preclinical study on SCD and bT

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.

11 Dec 2012

Sickle cell disease trial completes phase 3 patient enrollment

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.

5 Dec 2012

Novartis to highlight key data from extensive oncology portfolio at SABCS and ASH meeting

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.

30 Nov 2012

Acetylon's ective histone deacetylase inhibitors to be showcased at 54th ASH annual meeting

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.

21 Nov 2012

Sickle cell disease treatments: An interview with Dr Niihara, CEO of Emmaus Medical

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.

23 Oct 2012

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

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).

3 Oct 2012

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

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.

20 Sep 2012

Data from Sangamo BioSciences’ SB-728-T Phase 1 clinical programs on HIV/AIDS

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).

11 Sep 2012

bluebird bio completes $60 million Series D financing

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.

25 Jul 2012

Mechanical treatment shows promise in thalassemia

Vibration therapy may be an effective nonpharmacologic intervention to increase bone mass in patients with thalassemia, US researchers report

10 Jul 2012

Treating Sickle Cell Disease with L-Glutamine

By focusing on how best to help patients, researchers can sometimes find solutions that are much simpler than anticipated. That has been the guiding approach to our efforts to develop a treatment for sickle cell disease.

3 Jul 2012

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

bluebird bio, a leader in the development of innovative gene therapies for severe genetic disorders, announced today that both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have granted an orphan drug designation to its investigational gene therapy product for the treatment of adrenoleukodystrophy (ALD).

19 Jun 2012

Third Rock Ventures announces formation of Global Blood Therapeutics

Third Rock Ventures, LLC today announced the formation of Global Blood Therapeutics, Inc. and its $40.7 million Series A financing of the company.

15 Jun 2012

Alnylam first quarter revenues decrease to $20.6 million

Alnylam Pharmaceuticals, Inc., a leading RNAi therapeutics company, today reported its consolidated financial results for the first quarter 2012, and company highlights.

4 May 2012

New gene transfer technique can treat beta-thalassemia, sickle cell anemia

A team of researchers led by scientists at Weill Cornell Medical College has designed what appears to be a powerful gene therapy strategy that can treat both beta-thalassemia disease and sickle cell anemia. They have also developed a test to predict patient response before treatment.

28 Mar 2012

Shire enters agreement to acquire FerroKin BioSciences

Shire plc, the global specialty biopharmaceutical company, announces that it has signed an agreement to acquire FerroKin BioSciences, Inc., for an upfront payment of $100 million, payable in cash at closing, plus potential post-closing milestone payments of up to $225 million, depending upon the achievement of certain clinical development, regulatory and net sales targets.

15 Mar 2012

Thalassemia News and Research

Latest Thalassemia News and Research

Resonance Health receives FDA marketing authorization for FerriScan R2-MRI device

Acceleron commences ACE-536 phase 2 study in myelodysplastic syndromes

Novartis receives FDA approval for Exjade to treat chronic iron overload in NTDT patients

Penn Medicine to establish new center for treatment and research of blood diseases

Acetylon announces results from selective HDAC 1/2 inhibitor preclinical study on SCD and bT

Sickle cell disease trial completes phase 3 patient enrollment

Novartis to highlight key data from extensive oncology portfolio at SABCS and ASH meeting

Acetylon's ective histone deacetylase inhibitors to be showcased at 54th ASH annual meeting

Sickle cell disease treatments: An interview with Dr Niihara, CEO of Emmaus Medical

Haploidentical bone marrow transplants for sickle cell disease: an interview with Dr. Javier Bolaños Meade

Enrollment complete in Gamida Cell’s NiCord Phase I/II trial for hematological malignancies

Data from Sangamo BioSciences’ SB-728-T Phase 1 clinical programs on HIV/AIDS

bluebird bio completes $60 million Series D financing

Mechanical treatment shows promise in thalassemia

Treating Sickle Cell Disease with L-Glutamine

FDA, EMA grant orphan drug designation to bluebird bio's gene therapy product for adrenoleukodystrophy

Third Rock Ventures announces formation of Global Blood Therapeutics

Alnylam first quarter revenues decrease to $20.6 million

New gene transfer technique can treat beta-thalassemia, sickle cell anemia

Shire enters agreement to acquire FerroKin BioSciences

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

Thalassemia News and Research

Latest Thalassemia News and Research

Small solutions for big problems in drug discovery and delivery

Professor Nancy Ip: Pioneering New Paths in Neurodegenerative Therapy

Utilizing process Raman in optimizing cultivated meat production

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