April 30, 2013 – The medical journal Pediatric Blood and Cancer published online on April 23 a study entitled “The Palatability and Tolerability of Deferasirox Taken With Different Beverages or Food.” This is known to many in the thalassemia community as “the Exjade food study;” as the official title suggests, it looks at the option […]
March 28, 2013 – Shire, a specialty biopharmaceutical company, is currently in clinical trials with a new iron chelator. Below, Shire responds to questions from CAF concerning the status of the drug, currently known as SPD602. How did Shire become interested in SPD602? In April 2012, Shire acquired FerroKin BioSiences, Inc. and the iron chelator […]
February 22, 2013 – Funding for the National Institutes of Health (NIH) is in serious jeopardy. Without congressional action, NIH will receive a $1.6 billion cut when sequestration begins on Friday, March 1. It is critical that all Representatives and Senators hear from patients about the impact that inadequate funding has on the development of […]
January 28, 2013 – CAF is pleased to share the information from selected presentations from the 2012 CAF Patient~Family Conference. We are posting here the presentation on “Iron Levels: How Low Can You Go?” More videos will be posted as they become available. Stay tuned for information on the 2013 Patient~Family Conference. It will be […]
January 28, 2013 – Acceleron, a biopharmaceutical company based in Cambridge, Massahusetts, in collaboration with Summit, New Jersey-based pharmaceutical partner Celgene Corporation, is developing two programs, sotatercept (formerly called ACE-011) and ACE-536, for the treatment of beta-thalassemia. Sotatercept and ACE-536 are biologic drugs that increase hemoglobin and red blood cells levels by promoting the maturation […]
January 23, 2013 – From an FDA press release: The U.S. Food and Drug Administration today expanded the approved use of Exjade (deferasirox) to treat patients ages 10 years and older who have chronic iron overload resulting from a genetic blood disorder called non-transfusion-dependent thalassemia (NTDT). NTDT is a milder form of thalassemia that does […]
January 22, 2013 – The Thalassaemia International Federation (TIF) has scheduled two concurrent international conferences for October 20-23, 2013 in Abu Dhabi in the United Arab Emirates. Information about the 13th International Conference on Thalassemia and Hemoglobinopathies and the 15th International Conference for Parents and Patients can be found here. The website for the Conferences states that […]
January 10, 2013 – CAF has learned that the first U.S.-based clinical trial in gene therapy for beta-thalassemia has begun treating trial participants. The Phase 1 study, the official title of which is “ß-Thalassemia Major With Autologous CD34+ Hematopoietic Progenitor Cells Transduced With TNS9.3.55 a Lentiviral Vector Encoding the Normal Human ß-Globin Gene,” is […]
January 8, 2013 – The 1st Scientific Meeting of THALAMOSS will be held on January 14, 2013 at Ferrara University in Italy. The theme of the meeting is “From basic research to novel applications on the road of personalzied treatment of thalassemia.” THALAMOSS stands for THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassemia. According […]
November 21, 2012 – CAF is pleased to share the information from selected presentations from the 2012 CAF Patient~Family Conference. We are posting here the presentation on “Management of the Non-Transfused Patient.” More videos will be posted as they become available. Keep an eye out for information on the 2013 Patient~Family Conference. We will post […]
