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Summary of Edition of 23 April 2024

EU Parliament adopts position on pharmaceutical reforms at plenary session

The European health data sharing landscape: towards FAIRness for rare disease screening

ERN TransplantChild: Registration open for 5th workshop

ERKNet: New clinical practice recommendations published

ERN BOND survey on AI use in diagnostics

Register now to join Orphanet at ECRD 2024!

Implementing single-cell RNA sequencing in low- and middle-income countries: Towards a more diverse genomic research

Risk sharing agreements in federalised health systems: Lessons from the Canadian Fabry Disease Initiative

Survey on rare disease management and digital health

Rare Voices Australia's Board of Directors is recruiting

National workshops on patients' rights and rare diseases: Malta recap

2024 updated EMA fees

ACT EU launches multi-stakeholder platform

EJP RD: New course on early phase trial designs in rare diseases

Trends in orphan designations for paediatric rare neurological diseases

40 years of rare cancer drug approvals in the United States: Progress since the Orphan Drug Act

EMA: No positive opinions on market authorisation with an orphan designation in March 2024

EMA: Six new positive opinions for orhan designation accorded by the COMP in March 2024

FDA: Thirteen new market authorisations granted in March 2024

FDA: Thirty-eight new orphan designations approved in March 2024

Consensus guidelines for comprehensive phenotyping of ultra-rare genetic disorders

Federated data networks for increased transparency in real-world evidence studies

How trustworthy is ChatGPT when it comes to rare diseases? Evidence from sarcoma

Estimating prevalence and incidence of lymphangioleiomyomatosis using national administrative databases

Credits

OrphaNews, The Newsletter for the Rare Diseases Community.
The production of OrphaNews is supported by the Fondation IPSEN – Fondation de France.
Editor-in-chief: Ana Rath
Associate Editor: Charlotte Rodwell
Editor: Madeline Cuillerier
Editor for Scientific Content: Henri Jautrou
Scientific monitoring: Hélène Jagline
Contact Us
Editorial Board: Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Anna Bucsics, Ivana Cattaneo, Daria Julkowska, Alexis Arzimanoglou, Henri Jautrou, Holm Graessner, Julie Bruyere-Zrelli, Andrea Osvoll, Stanislav Ostapenko, Valentina Bottarelli, Dave Pearce, Samantha Parker, Alexandra Heumber Perry

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.