Back

Summary of Edition of 30 May 2018

Shaping the future for rare diseases : back from ECRD2018

Welcome to the new Editor-in-Chief of OJRD!

Orphanet Polish is here!

Orphanet has released Spanish translations of its Emergency Guidelines and Disability factsheet

FDA finalizes guidances to accelerate the development of reliable NGS tests

EURORDIS Black Pearl Awards: Nominate a rare disease star

Blockchain to enable medical data to be stored and transmitted safely and effectively

Genomic sequencing tests: the evolution of coverage policies

Affordability and availability of off-patent drugs in the US

Orphan medicines availability for outpatient care in 24 European countries

Report of the ISPOR rare disease special interest group on the challenges faced by HTA and research on rare disease

Gene therapy in rare diseases: the benefits and challenges for Strimvelis in ADA-SCID

SHARE initiative: collaborative paediatric research including biobanking

ERN RARE-LIVER new opportunity for rare liver patients

Credits

OrphaNews, The Newsletter of the Rare Diseases Community.
OrphaNews is supported by the European Commission's DG SANTE (RD-ACTION Joint Action N° 677024) and the French Muscular Dystrophy Association (AFM)
Editor-in-chief: Kate Bushby, Ana Rath
Editor:  Déborah Deprez, Divya Unni
Editor for Scientific Content: Sophie Höhn, Déborah Deprez
Contact Us
Editorial Board: Valentina Bottarelli, Teresinha Evangelista, Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Domenica Taruscio, Ariane Weinmann

Advisory Editorial Board: Ségolène Aymé, Anna Bucsics, Paul Boom, Bruno Dallapiccola, Jordi Llinares-Garcia, Alastair Kent, Milan Macek, Till Voigtländer

INTERNATIONAL CORRESPONDENTS
Orphanet Partner Country Representatives: Romi Armando (Argentina), Kristine Hovhannesyan (Armenia), Hugh Dawkins (Australia), Till Voigtlander (Austria), Rumen Stefanov (Bulgaria), Micheil Innes (Canada), Ingeborg Barisic (Croatia), Violetta Anastasiadou (Cyprus), Milan Macek (Czech Republic), John Rosendahl Ostergaard (Denmark), Vallo Tillmann (Estonia), Sirpa Ala-Mello (Finland), Joerg Schmidtke (Germany), Eileen Treacy (Ireland), Annick Raas-Rothschild (Israel), Bruno Dallapiccola (Italy), Tomoko Kodama (Japan), Jana Lepiksone (Latvia), André Mégarbané (Lebanon), Vaidutis Kucinskas (Lithuania), Yolande Wagener (Luxembourg), Abdelaziz Sefiani (Morocco), Gert-Jan van Ommen (Netherlands), Stein Are Aksnes (Norway), Malgorzata Krajewska-Walasek (Poland), Paul Nogueira (Portugal), Cristina Rusu (Romania), Dragica Radojkovic (Serbia), Laszlo Kovacs (Slovakia), Luca Lovrecic (Slovenia), Francesc Palau (Spain), Désirée Gavhed (Sweden), Loredana D'Amato Sizonenko (Switzerland), Dorra H'mida (Tunisia), Ugur Ozbek (Turkey), Sarah Stevens (UK) 
Orphanet - All rights reserved


Disclaimer: This newsletter is part of the project / joint action .677024 / RD-ACTION. which has received funding from the European Union.s Health Programme (2014-2020).
The content of newsletter represents the views of the Editorial Board only and is his/her sole responsibility; it cannot be considered to reflect the views of the European Commission and/or the Consumers, Health, Agriculture and Food Executive Agency or any other body of the European Union. The European Commission and the Agency do not accept any responsibility for use that may be made of the information it contains.

 

Photo credit : Serimedis (unless otherwise stated)