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Summary of Edition of 06 October 2025

Three new national plans for rare diseases

EURORDIS launches new Rare Barometer survey on what helps

ERNICA & eUROGEN launch Flagship Surgical Training Programme

EuroBloodNet Thursdays Webinars accredited by EBAH

ERKNet: Ongoing disease-specific surveys

Recent articles and resources published by ERNs

2024 Activity Report available now!

University of Geneva course introduces another cohort of students to rare diseases

WHA Resolution on Rare Diseases: Moving from adoption to implementation

Australia: RVA unveils report on Australia's Top 10 Rare Disease Research Priorities

New Zealand: Participate in the RDNZ survey on living with a rare condition

USA: FDA introduces new Rare Disease Evidence Principles

USA: NORD introduces new online course on data literacy

TREAT-NMD: New Family Guide for Limb-Girdle Muscular Dystrophy

TREAT-NMD survey on educational programming for 2026

USA: C-Path International Neonatal Consortium survey

Healthcare professionals & researchers: Share how you use patient experience data

EURORDIS Open Academy 2026: Applications open!

EURORDIS: Virginie Bros-Facer to step down as CEO

Outcomes of the EMA medicines repurposing pilot

JARDIN: New video tutorial on care pathways for patient representatives

TEHDAS2 publishes guideline on data use in secure processing environments

How are orphan drug prices determined in South Korea? An analysis of reimbursement approvals from 2012-2022

EMA: Three new market authorisations with an orphan designation in July 2025

EMA: Nineteen new orphan designations accorded by the COMP in July 2025

FDA: Eight new market authorisations with orphan designation granted in July 2025

FDA: Thirty-four new orphan designations approved in July 2025

Improving detection and treatment of rare diseases within psychiatry

Undiagnosed Hackathons: Ending the diagnostic odyssey through multidisciplinary collaboration

Prenatal diagnosis in India: Fourteen years' of results from a genetic centre

Can cognitive behavioural group therapy help people living with a rare disease?

MINDDS-Connect: How federated data platforms protect privacy & promote research

Epidemiology of rare diseases in China

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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 Bruyère-Zrelli, Julien Poulain, Valentina Bottarelli, Dave Pearce, Samantha Parker, Alexandra Heumber Perry, Perrine Renard

ADVISORY EDITORIAL BOARD
Orphanet Partner Country Representatives: Till Voigtlander (Austria), Annabelle Calomme (Belgium), Rumen Stefanov (Bulgaria), Marios Antoniades (Cyprus), Milan Macek (Czech Republic), Vallo Tillmann (Estonia), Helena Kääriäinen (Finland), Oleg Klivdize (Georgia), Carina Thomas (Germany), Eleftherios Thireos (Greece), Eileen Treacy (Ireland), Annick Raas-Rothschild (Israel), Bruno Dallapiccola (Italy), Atsuhiko Kawamoto (Japan), Gulnara Salavatovna Svyatova (Kazakhstan), Madara Auzenbaha (Latvia), Birute Tumiene (Lithuania), Silvana Masi (Luxembourg), Dijana Plaseska Karanfilska (North Macedonia), Abdelaziz Sefiani (Morocco), Wendy va Zelst-Stam (Netherlands), Stein Are Aksnes (Norway), Krystyna Chrzanowska (Poland), Carla Pereira (Portugal), Cristina Rusu (Romania), Maja Stojiljkovic (Serbia), Luca Lovrecic (Slovenia), Francesc Palau (Spain), Rula Zain (Sweden), Loredana D'Amato Sizonenko (Switzerland), Dorra H’mida-Ben Brahim (Tunisia), Ugur Ozbek (Turkey)

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.