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Summary of Edition of 08 July 2025

World Health Assembly adopts first-ever Resolution on rare diseases

Endo-ERN & ERN BOND: Apply for a EuRREB participating centre voucher

Endo-ERN: Recap of the 2025 General Assembly

ERN-RND: Winter School 2025 recordings now available

Recent scientific publications by ERNs

Orphadata.com & ORPHAcode.org: Two new sites to improve access to Orphanet data

Orphanet-AISBL officially launches with first General Assembly

Version 1.0 of the TREAT-NMD sNMD Dataset now available

Reflecting on 15 years of work to improve health equity for Aboriginals living with a rare disease in Western Australia

European hearing informs rare disease research and policy activities

ERDERA survey on patient involvement in research

New EMA & HMA joint workplan on data and AI in medicines regulation

Screen4Care publishes TREAT genomic NBS panel

ELIXIR releases new international strategy and strategic documents

New Solve-RD findings on approaches to solving undiagnosed rare disease cases

Apply for the HTA Regulation capacity-building programme

Recent events at the European level

Understanding the cost of the diagnostic odyssey for rare diseases: Findings from Germany

Making rare disease clinical trials more accessible: Current barriers and recommendations

EMA: New market authorisations with an orphan designation in April and May 2025

EMA: New orphan designations accorded by the COMP in April and May 2025

FDA: New market authorisations with orphan designation granted in April and May 2025

FDA: New orphan designations approved in April and May 2025

Post-diagnosis needs of parents in rare disease families: Findings from Japan's Initiative on Undiagnosed and Rare

Non-professional caregiving, health-related quality of life and productivity: Real-world findings

EJP RD findings on addressing methodological issues in rare disease clinical trials

Using new technologies to link rare diseases with common ones

Introducing: More to Explore!

Credits

OrphaNews, The Newsletter for the Rare Diseases Community.
Editor-in-chief: Ana Rath
Associate Editor: Charlotte Rodwell
Editor: Madeline Cuillerier
Scientific editor: Perrine Renard
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Editorial Board: Victoria Hedley, Charlotte Rodwell, Anna Bucsics, Ivana Cattaneo, Daria Julkowska, Alexis Arzimanoglou, Holm Graessner, Julie Bruyere-Zrelli, Andrea Osvoll, Stanislav Ostapenko, Valentina Bottarelli, Dave Pearce, Samantha Parker, Alexandra Heumber Perry, Perrine Renard

ADVISORY EDITORIAL BOARD
Orphanet Partner Country Representatives: Romi Armando (Argentina), Tamara Sarkisan (Armenia),  Till Voigtlander (Austria), Elfriede Swinnen (Belgium), Rumen Stefanov (Bulgaria), Ingeborg Barisic (Croatia), Marios Antoniades (Cyprus), Milan Macek (Czech Republic), Vallo Tillmann (Estonia), Helena Kääriäinen (Finland), Stefanie Weber (Germany), Eileen Treacy (Ireland), Annick Raas-Rothschild (Israel), Bruno Dallapiccola (Italy), Atsuhiko Kawamoto (Japan), Madara Auzenbaha (Latvia), Birute Tumiene (Lithuania),  Dijana Plaseska Karanfilska (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), Gabriela Hrčková (Slovakia), Luca Lovrecic (Slovenia), Francesc Palau (Spain), Rula Zain (Sweden), Loredana D'Amato Sizonenko (Switzerland), Dorra H’mida-Ben Brahim (Tunisia), Ugur Ozbek (Turkey), Sarah Stevens (UK)

Country Correspondants: Gareth Baynam (Australia)
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 its financers.