Tag Archives: Featured

TIF’s Renzo Galanello Fellowship Programme is back again in 2020!

  FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical trial. Patient recruitment is open for the second part of the trial (NCT03815695). Information about trial locations, all in the U.S., is available here. ...

  The ‚FAiTh – Fight Against Thalassemia‘ group,  a longtime member of TIF operating in Pakistan, is dedicated to support, serve and love people with thalassaemia and their families, intending to create a general, massive awareness about the illness and motivate the public to further support thalassaemia patients. These are ...

  An investigational gene therapy for Sickle Cell Disease (SCD) and β-thalassaemia, known as ARU-1801, has been given Orphan Drug Designation an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The therapy is expected to increase functioning red blood cells through ...

  Chiesi Farmaceutici and Apotex (together with its American subsidiary, ApoPharma Usa), a company engaged in haematology and in the area of ​​neurodegenerative diseases, have announced that they have signed an important agreement, in which Chiesi has acquired worldwide rights for Deferiprone, an iron chelator indicated for the treatment of ...

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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, ...

  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 ...

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, ...

  Global Blood Therapeutics, Inc. announced only a few hours ago that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Oxbryta™ (previously known as Voxelotor) tablets for the treatment of sickle cell disease (SCD) in adults and children 12 years of age and older. Oxbryta, an oral ...