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Summary of Edition of 26 July 2018

RD Action Final Meeting: Legacies and future challenges

Third National Rare Disease Plan for France announced

OrphaNews introduces its new editor

Orphanet has released Spanish translations of its Emergency Guidelines

Orphanet has released Spanish translations of its Disability factsheets

New edition of Orphanet Report : List of rare diseases

New edition of Orphanet Report : Prevalence of rare diseases

Orphanet releases results of its annual users' survey

Rare diseases mentioned for the first time at WHO 71st World Health Assembly

China officially releases its first national list of rare diseases

Statement from FDA Commissioner on agency’s efforts to advance development of gene therapies

Luxembourg releases first national rare disease plan

Latvia Cabinet of Ministers approves draft regulation on “health care services in the field of rare diseases”

European Commission establishes Steering Group on Health Promotion, Disease Prevention and Management of

Stakeholders confirm support for strengthened EU cooperation on Health Technology Assessment (HTA)

The impact of Brexit on UK pharmaceutical sector

SE-ATLAS: Mapping RD expert centres in Germany

EU Health budget 2021-2027

New Joint-Action to tackle health inequalities

ORPHA codes: new guidance documents available for codification purposes

EURORDIS: Call for nominations for Black Pearl Awards

ELIXIR Annual Report 2017

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 April 2018

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 28-31 May 2018

Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 25-28 June 2018

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation for

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation for

Committee for Orphan Medicinal Products (COMP) meeting report on the review of applications for orphan designation for

New European marketing authorisation of medicine with orphan designation

New European marketing authorisation of medicine with orphan designation

New European marketing authorisation of medicine with orphan designation

New European marketing authorisation of medicine with orphan designation

New European marketing authorisation of medicine without orphan designation

EMA: Update on guidance and forms for orphan designation

FDA April 2018 marketing approvals of orphan drugs

FDA May 2018 marketing approvals of orphan drugs

FDA June 2018 marketing approvals of orphan drugs

FDA approves the first drug with an indication for treatment of smallpox

Policy options regarding US importation for sole-source medicines

RD-Connect, NeurOmics and EURenOmics: Collaborative European Initiative for Rare Diseases

Recent developments in clinical terminologies – SNOMED CT, LOINC and RxNorm

Newborn bloodspot screening National Policy Framework in Australia

NIH: "All of Us" program

Rare disease and mental health report

New call for proposals by the European Commission

Phenylketonuria Round Table at the EU Parliament

New ESMO-EURACAN guidelines

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: Ana Rath
Editor: Agathe Franck
Editor for Scientific Content: Agathe Franck
Contact Us
Editorial Board: Valentina Bottarelli, Teresinha Evangelista, Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Domenica Taruscio, Ariane Weinmann

Advisory Editorial Board: 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) 
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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)