Beta Thalassemia News and Research

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Beta-thalassemia (ß-thalassemia) is a form of thalassemia due to mutations in the HBB gene on chromosome 11 , inherited in an autosomal recessive fashion.
Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam releases positive top-line data from ALN-TTRsc Phase I trial for TTR-mediated amyloidosis

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam Pharmaceuticals presents new pre-clinical data of ALN-AT3 at ISTH meeting

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam presents new pre-clinical data of ALN-CC5 for treatment of complement-mediated diseases

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Alnylam reports pre-clinical data from ALN-AS1 program for treatment of AIP

Two studies shed light on molecular biology of three blood disorders

Two studies shed light on molecular biology of three blood disorders

Acceleron commences ACE-536 phase 2 study in beta-thalassemia

Acceleron commences ACE-536 phase 2 study in beta-thalassemia

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Acceleron commences ACE-536 phase 2 study in myelodysplastic syndromes

Acceleron commences ACE-536 phase 2 study in myelodysplastic syndromes

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

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

Sickle cell disease trial completes phase 3 patient enrollment

Sickle cell disease trial completes phase 3 patient enrollment

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

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

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

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

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

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

bluebird bio completes $60 million Series D financing

bluebird bio completes $60 million Series D financing

Treating Sickle Cell Disease with L-Glutamine

Treating Sickle Cell Disease with L-Glutamine

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

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

Alnylam first quarter revenues decrease to $20.6 million

Alnylam first quarter revenues decrease to $20.6 million

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

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

New gene editing technique can heal sickle cell patients with their own cells

New gene editing technique can heal sickle cell patients with their own cells