The third International Workshop “Rare disease and orphan drug registries” will be hold from 24th to 25th November, 2014 in Rome, Italy. The event is organized within the EPIRARE project. Among the main topics are: the contribution of registration activities to recent advancements in the medical history, epidemiology and pathogenesis of rare diseases as well as patient care and quality of life; practical and innovative applications of registries; lessons learned in the management of rare diseases registries; the impact of e-health initiatives, new communication technologies and social networks. The deadline for the submission of abstracst is 12th October 2014. For more information, please visit the official EPIRARE website.
Sixth International Meeting on Pulmonary Rare Diseases and Orphan Drugs will be held on 27-28 February 2015 in Milan, Italy. It is the only European event dedicated to different types of rare pulmonary diseases affecting both parenchymal and vascular structures. Exchange and dissemination of knowledge from experts in various departments of pulmonary medicine is part of the challenge, aimed at generating new ideas in science and clinical practice. These actions will be beneficial both for patients and for doctors, whose common goal is early diagnosis, treatment and care for people with rare lung diseases. For more information, please visit the official website of the event.
Traditionally, the National Alliance of People with Rare Diseases (NAPRD) will hold a meeting of patient representatives during the Fifth National Conference on Rare Diseases. The main topic of the event is the adoption and promulgation of Ordinance № 16 of 30 July 2014 on the terms and procedure for registration of rare diseases and centers of expertise and reference networks for rare diseases and what changes would face the patients as a result of this document. For more information and program of the meeting, please visit the official website of NAPRD.
Professor Haluk Topaloglu from the Hacettepe University School of Medicine, Ankara, Turkey, will be one of the keynote speakers at the 5-th National Conference for Rare Diseases and Orphan Drugs. Professor Topaloglu’s research interests include paediatric neuromuscular disorders, neurogenetics, and developmental aspects in child neurology. He is currently Turkey’s representative to the European Science Foundation’s (ESF) Medical Research Group (EMRC). Haluk has served as the secretary of the World Muscle Society (WMS) since 2002. He will provide a presentation on “Treatment efforts in Duchenne muscular dystrophy” at the conference. For more information, please visit the official website of the conference: www.conf2014.raredis.org.
The program of the 5th National Conference on Rare Diseases and Orphan Drugs, which will be held at the end of this week in Plovdiv, has been officially published. Among the main topics are: current trends in clinical trials for rare diseases, experience and good practices of clinical trials on rare diseases in Europe, value and implementation of clinical trials for rare diseases in Bulgaria and the EU, interaction between clinical trials and registries for rare diseases. For more information, please visit the official website of the event.
In 2014 Information Centre for Rare Diseases and Orphan Drugs (ICRDOD) celebrates its 10th anniversary respect and honors distinguished patients, doctors, scientists, journalists and politicians who have helped and contributed to progress in the overall care and approach to people with rare diseases in Bulgaria. ICRDOD is the first of its kind in Eastern Europe free educational and information service for rare diseases in support of patients and medical professionals. The awards will be officially presented at a special gala dinner on September 26, 2014 (Friday) during the 5th National Conference on Rare Diseases and Orphan Drugs. For more information, please visit the official website of the event.
Second European Conference on Aniridia will take place on 19th – 20th of September 2014 in Venice, Italy. The event is organized by Fondazione Banca degli Occhi del Veneto Onlus and English is the official language. The conference is aimed at medical professional, patients, health authorities. Among the main topics are: problems that patients and their relatives meet, Aniridia in pediatric age, European and international guidelines on Aniridia, the role of patients’ associations. For more information, please visit the official website of the event.
International Summer School on Rare disease and orphan drug registries will be launched next week in Rome, Italy. The event is organized by the Institute for Rare Diseases – Italy within the project EPIRARE (European Platform for Rare Disease Registries) and is intended for researchers, clinicians, policy makers, patients. During this training participants will learn the basic steps that must be taken in the establishment and management of patient registries for rare diseases according to the new technologies, new data collection and methods. Registration for participants is free and open to 12.09.2014 (Friday). For more information about the summer school, registration or using online streaming, please visit the official website of the Italian Institute for Rare Diseases.
The European Haemophilia Network has pubslished European Guidelines for the Certification of Haemophilia Centres. This document recommends establishing procedures for collaboration between the European Haemophilia Treatment Centres and the European Haemophilia Comprehensive Care Centres in order to ensure multidisciplinary and comprehensive patient care across Europe. Among the main recommendations are providing all the facilities that patients need, thorough information about each center, formalized working plan, describing the mission, objectives and policies of the centers. For more information, please visit the official website of the European Haemophilia Network.
The European Medicines Agency (EMA) has published an illustrated booklet that explains how the European regulatory system for medicines operates. It describes the way drugs are authorised and monitored in the European Union (EU) and how the European medicines regulatory network and the EMA work to ensure that patients in the EU have access to safe and effective medicines. For more information, please visit the official website of EMA.