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Summary of Edition of 28 April 2023

EMA strengthens PRIME scheme with new features

ERN-EuroBloodNet: Collaborating to overcome barriers in Sickle Cell Disease research

ERN-EuroBloodNet: New online educational program

Roundtable launch of the Global Nursing Network for Rare and Undiagnosed Diseases

Lessons learned for applicants seeking orphan designation in the United States

Australia: Moving towards a more sustainable rare disease workforce

India: Customs duties waived for rare disease drugs

EURORDIS and partners call for a more society-centred approach to the European Health Data Space

Translating research into innovation: Evidence from EJP RD

EPDOS.nl: A new website promoting open science

Gene-targeted therapies: Value, affordability, and access

Drug repurposing for rare diseases through academia-industry partnerships

EMA: No new positive opinions on Market Authorisation with an Orphan Designation in March 2023

EMA: Nine new positive opinions for orphan designation accorded by the COMP in March 2023

FDA: Nine new Market Authorisations granted in March 2023

FDA: Forty new orphan designations approved in March 2023

Research challenges and priorities in the inherited bleeding disorder community

Towards a regulatory framework for orphan medical devices

Using administrative databases to guide screening interventions for rare diseases: The case of Fabry disease in

Black women's experiences with noninvasive prenatal testing in the United States

Quality of life in the Latin American neuromuscular disease community during COVID-19

Overcoming challenges in rare disease trial design: Evidence from pantothenate kinase-associated neurodegeneration

Online registry building: Guidance for patient organisations

The Raresevoir: A new database for gene association from the 100,000 Genomes Project

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, Hélène Dollfus, Alexis Arzimanoglou, Henri Jautrou, Holm Graessner, Julie Bruyere-Zrelli

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.

Photo credit : Serimedis (unless otherwise stated)