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Summary of Edition of 03 June 2026

ECRD 2026: Rare Diseases in a Changing & Competitive Europe

2025 Activity Report leaflet available now!

Flagship ERN article published

2026 Call for ERN affiliated partners now open

ERN CRANIO: Survey on psychological services in craniofacial care

Recent articles and resources published by ERNs

Call to action to advance nursing education for rare diseases

Towards a Global Action Plan for Rare Diseases: Considerations for implementing the WHA resolution

New publications from IRDiRC

Participate in the survey on the OrphanAnesthesia platform

Australia: Rare Barometer factsheets provide insight into rare disease community's perspectives

Australia announces expansion of newborn screening for X-linked adrenoleukodystrophy

Australia: RVA responds to federal budget 2026-27

India announces new Centres of Excellence for rare disease

USA: FDA issues request for input on drug repurposing

European Action Plan for Rare Diseases gains momentum at Parliamentary event

Provisional agreement reached on Critical Medicines Act

EMA launches pilot to support breakthrough medical device development

EURORDIS names Jean Saslawsky as new CEO

Preliminary results from the ERDERA survey on consultancy service needs

EMA consultation on proof-of-concept data in paediatric oncology

TEHDAS2: Public consultation on draft guidance documents

HTACG adopts guiding principles on data transparency

Luxembourg: ALAN 2025 activity report available now

Costs associated with genome sequencing and bioinformatics for Indigenous children with a suspected rare disease

EMA: New market authorisations with an orphan designation in March 2026

EMA: New orphan designations accorded by the COMP in March 2026

FDA: New market authorisations with orphan designation granted in March 2026

FDA: New orphan designations approved in March 2026

Understanding barriers to genetic counselling for rare diseases in LMICs

Scoping review of ethical, legal and social challenges associated with genomic NBS

Psychological burden of rare diseases at an outpatient clinic in China

Mapping medical terminologies to analyse COVID-19 outcomes among rare disease patients

Exploring digital twin applications in paediatric rare disease research

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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.