EMA strengthens PRIME scheme with new features
ReadSummary of Edition of 28 April 2023
Editorial
Political news
International News
Roundtable launch of the Global Nursing Network for Rare and Undiagnosed Diseases
ReadLessons learned for applicants seeking orphan designation in the United States
ReadAustralia: Moving towards a more sustainable rare disease workforce
ReadIndia: Customs duties waived for rare disease drugs
ReadHealth Economics
Gene-targeted therapies: Value, affordability, and access
ReadOrphan Drugs
Drug repurposing for rare diseases through academia-industry partnerships
ReadEMA: No new positive opinions on Market Authorisation with an Orphan Designation in March 2023
ReadEMA: Nine new positive opinions for orphan designation accorded by the COMP in March 2023
ReadFDA: Nine new Market Authorisations granted in March 2023
ReadFDA: Forty new orphan designations approved in March 2023
ReadQuality of Life
Quality of life in the Latin American neuromuscular disease community during COVID-19
ReadStudy Design
Overcoming challenges in rare disease trial design: Evidence from pantothenate kinase-associated neurodegeneration
ReadRegistries and Biobanks
Online registry building: Guidance for patient organisations
ReadBioinformatics and Biotechnology
The Raresevoir: A new database for gene association from the 100,000 Genomes Project
ReadScientific news
And also...
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)