The primary aim of this study is to evaluate the effect of the drug N-Acetylcysteine on the frequency of pain in daily life in patients with Sickle Cell Disease (SCD).

Pain is an invalidating hallmark of this disease and has a considerable impact on the Quality of Life of patients and the medical health care system. Oxidative stress is hypothesized to play a central role in its pathophysiology. In pilot studies the administration of N-Acetylcysteine (NAC) resulted in a reduction of oxidative stress. Moreover, administration of NAC seemed to decrease hospitalization for painful crises in a small pilot study in patients with SCD.

This study will be performed as a multicenter, randomized, controlled trial where patients will be treated with either NAC or placebo for a period of 6 months. The investigators expect that NAC can reduce the frequency of pain in patients with SCD, thereby improving their quality of life and participation in society.


More information:, ITHANET Clinical Trials


The seventh meeting of the European Union Committee of Experts on Rare Diseases (EUCERD) took place on 31 January – 1 February 2013 in Luxembourg: the plenary session was preceded by breakout sessions by stakeholder group to discuss the draft EUCERD Recommendations on Common Principles and Consensus on patient registries and data collection for rare diseases.


Read the full summary here (PDF) and visit the official EUCERD website.

thal day2013

May 8 marks the international thalassaemia day aiming to raise awareness of the disease. On this special day, every year, Thalassaemia International Federation (TIF) focuses on a different theme that intervenes with the quality and appropriate treatment of patients and impacts their quality of life. This year TIF is bringing to the forefront the treatment around thalassaemia intermedia and is inviting all national thalassaemia associations across the world and other patient support groups to find out more about this disease, within the theme:

The right for quality health care of every patient with thalassaemia: major and beyond

For more information visit the dedicated International Thalassaemia Day at the TIF website and read the TIF's message.



The objective of this study is to evaluate the pharmacokinetics of deferiprone and its 3-O-glucuronide metabolite following administration of a single 1500 mg dose of Ferriprox in patients with sickle cell disease. The study, sponsored by ApoPharma, starts in May 2013 and the expected completion date is July 2013.


More information:, ITHANET Clinical Trials