The European Commission has today, on Rare Disease Day 2013, announced €144 million of new funding for 26 research projects on rare diseases. The projects will help improve the lives of some of the 30 million Europeans suffering from a rare disease. The selected projects bring together over 300 participants from 29 countries in Europe and beyond, including teams from leading academic institutions, SMEs and patients' groups. The goal is to pool resources and work beyond borders, to get a better understanding of rare diseases and find adequate treatments.

One of the projects, entitled THALAMOSS (THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassemia), aims to develop universal sets of markers and techniques for stratification of β-thalassaemia patients into treatment subgroups for (a) onset and frequency of blood transfusions, (b) choice of iron chelation, (c) induction of fetal hemoglobin, (d) prospective efficacy of gene-therapy.

To view all the projects, please see the MEMO/13/148 and visit the website. For more information, please see the official press release.

The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease. The study will also investigate reduction in daily pain in children who have sickle cell disease.

More information: clinicaltrials.gov, ITHANET Clinical Trials

The majority of school-age children with sickle cell disease (SCD) experience neurocognitive deficits, even in the absence of stroke. In particular, deficits in attention and working memory have emerged as two of the most common neurocognitive sequelae of SCD. Thus, the goal of the present proposal is to address feasibility and compliance of a novel computerized cognitive training program, Cogmed. Pilot data will also be collected to establish preliminary efficacy.

More information: clinicaltrials.gov, ITHANET Clinical Trials

This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case Sickle Cell Disease (SCD). In this research study, the investigators are specifically looking to see if Regadenoson is an effective treatment for pain crises and acute chest syndrome in SCD.

More Information: clinicaltrials.gov, ITHANET Clinical Trials