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Summary of Edition of 17 September 2019

The legacy of the Joint Action on Rare Cancers: Ten recommendations for the future policy on rare cancers in

New estimate of worldwide population affected by a rare disease at any point in time

Orphadata : New documents providing descriptions of datasets and tools

Japan: Elaboration of a data integration platform for rare disease research

China: Analysis of the social health care system as regards rare disease patients' needs

Canada: Possibilities of germline genome editing for rare diseases

Northern Ireland: Communication needs in Northern Irish healthcare system

Assessment of cost and efficacy of prenatal recall versus reflex DNA screening for trisomy 21, 18 and 13

Marketing of orphan drugs in China

Clinical pharmacology and rare disease research

Opinion on United States' recent decisions regarding the paediatric orphan drug market

Demonstrating not ineffectiveness, a two-stage approach for orphan drug review and approval
 

Israeli parents of children with Down Syndrome and non-invasive prenatal screening

New algorithm to assist physicians with the differential diagnosis of central ocular motor rare diseases

Human-on-a-chip technology for orphan drug development 

Quality of life in systemic sclerosis before and after autologous haematopoietic stem cell transplant

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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