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Summary of Edition of 29 July 2019

“Towards access of at least 1 million sequenced Genomes in the EU by 2022”

Orphanet improves its nomenclature and services for codification purposes

Impact of recent transformative therapies on global health systems 

Singapore’s new Rare Disease Fund

USA: NORD launches new grants and a new educational platform for rare diseases 

Australian Genomics: integrating genomics in federated healthcare

China: Extracting valuable data from Chinese hospitalisation summary reports

ERN Board of Member States decisions: more collaboration with industry and enlargement of the networks

E-REC: A new electronic reporting system

MetabERN: a survey on its research activities

Baroness Blackwood’s speech and new initiatives 

Nominations for the EURORDIS Black Pearl Awards 2020

Preferences regarding public funding of orphan drugs

Analysis of the Slovak extraordinary reimbursement regime

Analysis of factors for positive recommendations for rare disease drugs in Canada

Worldwide burden of musculoskeletal diseases

Patterns of research in rare disease orphan drugs development

Drug repurposing and drug databases

Benefits of and approaches for drug repurposing 

The CHMP recommended four medicines for approval at its May 2019 meeting

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 June 2019

Committee for Orphan Medicinal Products meeting report on the review of applications for orphan designation May 2019
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Committee for Orphan Medicinal Products meeting report on the review of applications for orphan designation June 2019

Meeting highlights from the Committee for Medicinal Products for Human Use 23-26 April 2019

DM-Scope registry and its benefits
 

Presentation of the Incontinentia Pigmenti Genetic Biobank project 

Analysing disease organ enables better genetics understanding

Survey findings on patient preferences regarding data sharing

Credits

OrphaNews, The Newsletter for the Rare Diseases Community.
OrphaNews is supported by the European Commission through the pilot projet 'Rare 2030 - a participatory foresight study policy-making rare diseases' (PP-1-2-2018-Rare 2030).
Editor-in-chief: Ana Rath
Associate Editor: Charlotte Rodwell
Editor: Florent Simon
Editor for Scientific Content: Claire Bernard
Scientific monitoring: Hélène Jagline
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Editorial Board: Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Anna Bucsics, Franz Schaefer, Ivana Cattaneo

ADVISORY EDITORIAL BOARD
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), Marios Antoniades (Chyprus), Milan Macek (Czech Republic), Vallo Tillmann (Estonia), Helena Kääriäinen (Finland), Susanne Morlot (Germany), Eileen Treacy (Ireland), Annick Raas-Rothschild (Israel), Bruno Dallapiccola (Italy), Atsuhiko Kawamoto (Japan), Ieva Malniece (Latvia), Birute Tumiene (Lithuania), Abdelaziz Sefiani (Morocco), Gert-Jan van Ommen (Netherlands), Stein Are Aksnes (Norway), Krystyna Chrzanowska (Poland), Mario Carreira (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-Ben Brahim (Tunisia), Sarah Stevens (UK)
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Disclaimer: 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)