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Orphanet website Ireland entry point

Orphanet Ireland
Orphanet international services
  • An inventory and classification of rare diseases
  • An encyclopaedia
  • A directory of services: clinics, laboratories, research projects, registries, clinical trials, patient organisations
  • An inventory of orphan drugs
  • Guidelines, reports
  • A newsletter
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Welcome to the Orphanet Ireland national website! 

 

This page presents the news, events and documents of national significance.
  • To register your activity click on "Register online" on the left tab
  • To provide an article regarding your activity contact idoia.gomez-paramio@manchester.ac.uk
  • To access all information on rare diseases, orphan drugs and expert services visit the main Orphanet portal (www.orpha.net).

 

 

News in Ireland

 

 

 

European AKU consortium launches first clinical study this spring 


This year the AKU Society will embark on international clinical trials for the drug nitisinone. Nitisinone is currently licensed for another rare disease but it has not yet been approved for treating patients with AKU. Previous findings have shown that nitisinone could be effective: clinical research in the US showed that levels of homogentisic acid (HGA), the molecule that causes all the damage in AKU, were reduced by up to 95%.

The first trial will take place at two centres: the Royal University Liverpool Hospital, UK, and Piestany, Slovakia. It will start in Spring and last four weeks. Patients will only need to visit their trial centre twice. Patients will be randomly divided into five groups that will each receive different doses of nitisinone. This is in order to determine the best dose of nitisinone in AKU patients to be used in a second, longer trial in late 2013. All travel costs will be covered for patients within Europe, with accommodation being provided at the trial centres.

If you are would like more information, please contact our Clinical Trials Coordinator, Hana Ayoob, on +44(0)1223 322897 or by email at hana@akusociety.org" target="_blank">hana@akusociety.org.

 

 

Save the date: The first International Rare Disease Research Consortium Conference in Dublin
 

 

The International Rare Diseases Research Consortium (IRDiRC) conference scheduled on 16-17 April 2013 in Dublin will be the premier venue for all rare disease stakeholders from around the globe to engage in stimulating discussions in an open environment that encourages you to think, learn, exchange views and network. This two day conference features keynote addresses and presentations by stalwarts in rare disease research from all over the world. Additionally, posters displaying results from research projects funded by IRDiRC members will be presented on Day 1. 

After its launch in April 2011, IRDiRC has garnered significant support around the globe. To begin with, three projects of the IRDiRC flagship venture have been granted around 40 million euros under the European FP7 plan. The goal of IRDiRC is to assist in delivering 200 new therapies for rare diseases and means to diagnose most rare diseases by the year 2020. The conference in Dublin will play a substantial role in advancing to these goals. 

Organised by the European Commission in association with the Irish presidency of the Council of the European Union, the IRDiRC conference registration is now open to the public. *
Register now to be a part of the IRDiRC conference
Learn more about IRDiRC 
 

 

 

News on Orphanet 

 

Orphanet has just released its mobile application!

 

 


Available on iPhone and compatible with iPad, the first version of the Orphanet application gives access to main services of Orphanet (encyclopaedia of rare diseases, expert centres and emergency guidelines) in English and French. A second version will soon be available in more languages (German, Italian, Spanish and Portuguese). The Orphanet application is freely downloadable on the Apple store. Please do not hesitate to contact us for any feedback on the application supportmobile.orphanet@inserm.fr" target="_blank">(supportmobile.orphanet@inserm.fr). *
 

 

 

Now available: comprehensive and updated list of orphan medicinal products


 

The latest addition to the Orphanet report series, Lists of medicinal products for rare diseases in Europeis now available. This exhaustive list has been compiled with the help of information on the European Medicines Agency and the DG health and consumers (DG Sanco) of the European Commission. The first part catalogues medicinal products with orphan drug designation that have been granted European marketing authorisation. It lists all the trade names of the medicinal products have been granted orphan designation in alphabetical order, along with date of market authorisation and the company holding the authorisation. The second part consists of authorised medicinal products that are intended to treat rare disease but do not have orphan designations. Notably, this list also underscores orphan medicinal products that have completed its 10 year market exclusivity period, allowing possible generics to enter the market. This report is also rich in other information such as the number of market authorisations for medicinal products per year as well as the number of authorisations within each ATC category. *
For more information on orphan medicinal products on the Orphanet website
For more information on orphan medicinal products on the EMA website

 

 

 

EUCERD unanimously adopts recommendation on European Reference Networks for Rare Diseases 


 

At their 7th Meeting on 31 January 2013 in Luxembourg, the European Union Committee of Experts on Rare Diseases (EUCERD) unanimously adopted their third recommendation to date, entitled 'EUCERD Recommendations on European Reference Networks for Rare Diseases'. 
The recommendations were elaborated by the Committee to feed into the work of the Cross-Border Healthcare Expert Group, currently in the process of aiding the European Commission implement the Cross-Border Healthcare Directive, adopted on 28 February 2011 (For further details). European Reference Networks (ERNs) are one of the structures foreseen by the Directive to share knowledge, facilitate the mobility of expertise, and to allow Member States to provide highly specialised services of high quality for patients where this would have been impossible without European networking, such as in the case of rare diseases. Member States are also encouraged in the Council Recommendation on an action in the field of rare diseases (8 June 2009) to help foster the participation of centres of expertise in these ERNs. The EUCERD has already elaborated recommendations concerning centres of expertise for rare diseases which describes how these centres could participate in such networks. 
The recommendation is addressed to the European Commission and the Member States and includes 21 individual recommendations covering a range of aspects including the mission, vision and scope of ERNs, their governance, their composition, their funding and evaluation, as well as their designation. 
The "EUCERD Recommendations on European Reference Networks for Rare Diseases" are now available here *

 

 

 

 

*Source: OrphaNews Europe

 

 

 

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Last update: 03/05/13




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