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Tag Archivi: Featured

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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TIF’s latest Newletter is out now!

Catch up with our most recent newsletter and get informed about TIF’S activities and achievements in the past few months! If you would also like to receive our news directly into your inbox, along with thousands of people across the world, subscribe here!

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LOVING LIFE ENOUGH by Mary Manachi

This is the story of the late Mrs. Mary Manachi from New Jersey, USA, who lost 3 children from thalassaemia back in the 50’s-60’s, when no drugs and medical treatments were available to help prevent their death at a fairly young age. Mrs. Manachi passed away a few years ago, ...

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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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