Tag archives : Patients

  Observed every year on the last day of February and in the 29th of February in 2020, Rare Disease Day seeks to raise awareness among the general public, as well as policymakers, public authorities, industry representatives, scientists, and health professionals about the more than 7,000 rare disorders that affect as many ...

  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 ...

The application period for the ‘Panos Englezos Prize 2020‘ is now open

  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 ...

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, ...

Written by Dinar Faiza (edited)   The Indonesian Thalassemia Foundation of Banyumas and the Banyumas District Government organised and launched, from November 1 to November 30, 2019, a massive pilot project of Thalassaemia Prevention Movement 2019, called  »Thalassaemia Month of Banyumas ». Its theme was entitled « Banyumas Goes To Zero Growth ...

  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 ...

  Victoria Gray, 34-year-old mother-of-four, has volunteered to be the first American to have their genes edited with the controversial CRISPR technology to treat her SickleCell disease (SCD). For the first time, doctors in the U.S. have used the powerful gene-editing technique CRISPR to try to treat a patient with a ...