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FT-4202 Granted FDA Fast Track, Rare Pediatric Disease Designations for SCD

FT-4202, a potential disease-modifying oral treatment for sickle cell disease being developed by Forma Therapeutics, has been given fast track and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). A fast track status is meant to accelerate the development and review of the treatment, facilitating discussions with the FDA and enabling the therapy ...

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Bluebird bio announces 1st commercial launch of ZYNTEGLO™in Germany

  Bluebird bio, Inc. announced earlier today the launch  of ZYNTEGLO™ in Germany,  the company’s one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related ...

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Deferasirox Accord: A new generic of Exjade recommended for approval by the EMA

The Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency‚ (EMA) committee responsible for human medicines, has recently adopted a positive opinion recommending the granting of a marketing authorisation for the medicinal product Deferasirox Accord, by Accord Healthcare S.L.U.   Deferasirox Accord is a generic of Exjade, ...

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Deferasirox Mylan: A new generic drug for the treatment of iron overload in β-thalassaemia major obtained approval by the EMA

  The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product “Deferasirox Mylan“, intended for the treatment of chronic iron overload due to blood transfusions in patients with β-thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias. The manufacturer of this medicinal product is Mylan S.A.S. “Deferasirox Mylan“ will be ...

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As survival increases in β-thalassemia, renal complications emerge

  In a scientific paper recently published in Hematology, researchers discussed the most common pathophysiologic and clinical manifestations of renal disease in patients with beta thalassaemia. “In recent years, the life expectancy of patients with thalassaemia has increased, and this has allowed previously unrecognized renal complications to reveal themselves,” said ...

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Biotech companies issue first joint declaration on human gene editing

Companies involved in human gene editing have entered the ethical debate about the controversial technology, issuing a joint declaration that DNA must not be altered in a way that passes genetic changes on to future generations. The Alliance for Regenerative Medicine (ARM), an international group representing the cell and gene ...

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