The Cooley's Anemia Foundation (CAF) sponsors incentive awards for thalassaemia patients to further their education and career goals and to help them live positively with thalassaemia. Applicants must be U.S. residents who are currently pursuing education in a graduate, undergraduate, associate, certificate or vocational level.

The incentive awards are given as follows:

·$2,000 for Doctorate programmes with the possibility of receiving two awards during the course of study.

·$1,500 for Master degree programmes with the possibility of receiving two awards during the course of study.

·$1,000 for Baccalaureate degrees with the possibility of receiving four awards during the course of study.

·$750 for an Associates degree, limited to two awards per applicant.

·$500 for certificate programmes lasting for one year or longer, limited to two awards per applicant.

·$250 for six-month certificate programmes or vocational training programmes, limited to two awards per applicant.

The application deadline is February 15, 2018.


More information: CAF announcement 




The Cooley’s Anemia Foundation is accepting applications for medical research grants and fellowships in areas related to thalassaemia. The awards are in 3 categories:

1. Support for Ongoing Clinical Research in Thalassaemia (Deadline: January 15, 2018 for a letter of intent and February 5, 2018 for invited full applications).

The goal of this initiative is to support investigators from all disciplines and backgrounds (MD, RN, PhD, MPH, MSW or other disciplines) with their ongoing clinical projects to address one or more of the following areas impacting patients with thalassaemia, including but not limited to: cardiac issues and iron overload; fertility, pregnancy and family planning; quality of life, psycho-social impact and/or burden of disease. Funding: $40,000 per year.

2. Clinical Trials in Thalassaemia Cell and Gene Therapy (Deadline: February 5, 2018).

This initiative aims to support clinicians and researchers involved with clinical applications of cell and gene therapy as to advance a cure for thalassaemia. Both phase I (safety) and phase II (efficacy) trials are eligible for support. Funding: $60,000 per year.

3. Research Fellowships (Deadline: February 5, 2018).

This initiative accepts applications from junior faculty and postdoctoral fellows involved with basic and translational research towards the understanding and/or treatment of thalassaemia and/or a complication that is related to thalassaemia. The areas of interest include, but are not limited to, studies of globin gene regulation, globin gene transfer and expression, fetal haemoglobin production, haematopoietic stem cell research, bone marrow transplantation, iron chelation and iron overload, endocrine and cardiac disorders in thalassaemia, and transfusion therapy and its complications.

More information: CAF announcement

fda logo

The U.S. Food and Drug Administration (FDA) approved Endari [PDUFA date: July 7, 2017] for the treatment of sickle cell disease (SCD). This marks a significant milestone in the field as Endari is the first and only FDA-approved treatment for pediatric patients and the first treatment in almost 20 years for adult patients. FDA approval was based on safety and efficacy data from a randomized, double-bind, placebo-controlled multi-centre clinical trial [NCT01179217] involving 230 adult and pediatric patients (5 to 58 years of age). Endari treatment improved the course of SCD, resulting in fewer sickle cell crises and hospitalizations, as well as a lower incidence of the life-threatening acute-chest syndrome. No major adverse events related to treatment were reported.

Endari is an orally-administered pharmaceutical grade L-glutamine made by Emmaus Life Sciences Inc. (Torrance, Calif.). It has received Orphan Drug designation in the U.S., Orphan Medicinal Product designation in the EU and Fast Track designation from the FDA.


More information: Press announcement

Biol. of Blood and Marrow Transpl






Allogeneic haematopoietic stem cell transplantation (HSCT) is the only established curative therapy for β-thalassaemia. Patients who opt for this treatment are faced with potential late effects that may lead to chronic health conditions. Long-term follow-up after HSCT has the potential to identify late complications of HSCT, allowing for implementation of standardized surveillance strategies and interventions to optimize outcomes and further increase survival rates. While long-term outcomes of HSCT for β-thalassaemia have been reported in literature, these are scarce and limited to single center experiences. An article published in the June 13 issue of the Biology of Blood and Marrow Transplantationreports late effects and long-term health among 176 pediatric patients affected by β-thalassaemia who survived 1 year and beyond after HSCT with a median follow-up of 7 years. This is an original report on long-term follow-up after HSCT for β-thalassaemia using data from multiple international transplant centres. Outcomes of interest included engraftment, chimerism, graft rejection, graft-vs-host disease, ferritin, growth velocity and measures of organ function. Although the study reported high survival among transplant recipients, late organ toxicity and growth impairment was frequently described in those greater than 7 years of age. The incidence of late effects highlighted the need of uniform, careful and systematic follow-up care as to correctly identify and promptly treat any complications that may arise after HSCT. The study also highlighted the need to systematically document the long-term outcomes of HSCT in data registries in order to optimize the tracking and management of post-HSCT health among transplant survivors.

More information:  Publication

irdirc logo

The International Rare Disease Research Consortium (IRDiRC) was launched in April 2011 at the initiative of the European Commission and the U.S. National Institutes of Health to foster international collaboration and maximize research output in the rare diseases (RD) field. The IRDiRC 2010-2020 goals are to develop 200 new therapies and the means to diagnose most RD by the year 2020. With IRDiRC's first goal completed ahead of time and the second being in close reach, the 3rd IRDiRC conference held in Paris in February 2017 celebrated these achievements and dedicated time to reflect on new goals towards better diagnosis and therapy for RD patients.

The overarching goals of IRDiRC for the upcoming decade have now been announced. These include:

  • 1) All patients coming to medical attention with a suspected (and known) RD will be diagnosed within one year. Undiagnosed patients will enter a globally coordinated diagnostic and research pipeline.
  • 2) 1000 new therapies will be approved, the majority of which will focus on RD without approved options.
  • 3) Methodologies will be developed to assess the impact of diagnoses and therapies on RD patients.
  • More information: IRDiRC News, IRDiRC Press Release3rd IRDiRC Conference-ParisRD-Connect News