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Summary of Edition of 04 April 2024

JARDIN: A new Joint Action to shape the future of ERNs

New amendments to EU pharmaceutical reform approved

New publication: ERKNet/ERA CPR for kidney involvement in tuberous sclerosis complex

ERKNet/EURORDIS joint webinar on patient involvement in guidelines

Apply to join IRDiRC's Regulatory Scientific Committee

APARDO and the rare disease landscape in Asia Pacific

Fulfilling unmet need in rare diseases in Latin America: the ERCAL initiative

Genetic Alliance UK publishes annual policy report

Genetic Alliance UK shares "Manifesto for rare diseases"

Political agreement reached on the European Health Data Space

EJP RD virtual platform seeking feedback

HTA, pricing and reimbursement of orphan drugs in Canada

What role for real-world evidence in FDA orphan drug approvals?

Looking back on the first 10 years of the FDA's priority review voucher program for paediatric diseases

GENets: How Denmark established expert collaboration networks for genetics

New recommendations for managing rare neurological diseases

NBS landscape in South Africa: current status and future prospects

Presenting the Turner Syndrome Research Registry

The HIBAD project: GDPR-compliant data sharing for clinical research

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.