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Summary of Edition of 09 November 2020

Update on the EU4Health Programme : ongoing negotiations

ERN-BOND: Implementation of the EuRR-Bone registry the COVID-19 Helpline for Rare Bone Diseases

EJP RD Call for Research Mobility Fellowships for ERN young researchers

EuroBloodNet: More webinars online

VASCERN: Six new languages added to the video on Klippel-Trenaunay syndrome

Orphanet Encyclopedia: 69 texts revised with European Reference Networks

China: A study on the need to empower rare diseases organisations

Evaluation of the EU Orphan Regulation

EJP RD partners to foster rare diseases clinical trial methodologies in small populations

EMA’s guideline on registry-based studies

Japan: Medical exome analysis and health‐economic outcomes

Assessment of FDA clinical trials grants on orphan products: outcomes and impact

Characteristics of orphan drug designation in Europe

Analysis of orphan drugs approved and reimbursed in Spain from 2003 to 2019

Establishing the reasonable price of an orphan drug

A Review of Submissions to the Canadian Agency for Drugs and Technologies in Health Common Drug Review

Undiagnosed Diseases Network and missed diagnoses: Clinically relevant lessons and resolved medical mysteries

Orphan drugs Registries: marketing tools or generating evidence

Open science in rare diseases

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: Josiane Kijigo
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, Irene Mathijssen, Daria Julkowska, Franz Schaefer, Ivana Cattaneo

ADVISORY EDITORIAL BOARD
Orphanet Partner Country Representatives: Romi Armando (Argentina), Kristine Hovhannesyan (Armenia), Till Voigtlander (Austria), Elfriede Swinnen (Belgium), Rumen Stefanov (Bulgaria), Micheil Innes (Canada), Ingeborg Barisic (Croatia), Marios Antoniades (Cyprus), Milan Macek (Czech Republic), John Oestergaard (Denmark), Vallo Tillmann (Estonia), Helena Kääriäinen (Finland), Susanne Morlot (Germany), Helen Michelakakis (Greece), Eileen Treacy (Ireland), Annick Raas-Rothschild (Israel), Bruno Dallapiccola (Italy), Atsuhiko Kawamoto (Japan), Ieva Malniece (Latvia), André Mégarbane (Lebanon), Birute Tumiene (Lithuania), Dijana PlaseskaKaranfilska (North Macedonia), Neville Calleja (Malta) Abdelaziz Sefiani (Morocco), Wendy va Zelst-Stam (Netherlands), Stein Are Aksnes (Norway), Krystyna Chrzanowska (Poland), Cristina Rusu (Romania), Sergey Kutsev (Russia), Dragica Radojkovic (Serbia), Laszlo Kovacs (Slovakia), Luca Lovrecic (Slovenia), Gabriela Hrčková (Slovakia), Francesc Palau (Spain), Rula Zain (Sweden), Loredana D'Amato Sizonenko (Switzerland), Dorra H’mida-Ben Brahim (Tunisia), Ugur Ozbek (Turkey), 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)