Looking back at Rare Disease Day 2023
ReadSummary of Edition of 15 March 2023
Editorial
Political news
Orphan Drugs
Current and future therapeutics for paediatric genetic diseases
Read
EMA: One new positive opinion on Market Authorisation with an Orphan Designation in February 2023
Read
Sixteen new positive opinions for orphan designation accorded in February 2023 by the COMP
ReadSeven new Market Authorisations granted in February 2023 by the FDA
ReadTwenty-three new orphan designations approved in February 2023 by the FDA
ReadQuality of Life
Quality of life and other social effects on people living with rare skin diseases
ReadStudy Design
Challenges in studying rare diseases of epigenetic origin
ReadRegistries and Biobanks
RareData EU: Guidance for real world data studies and building registries
ReadBioinformatics and Biotechnology
Considerations for adopting whole genome sequencing in paediatrics
ReadScientific news
And also...
Credits
OrphaNews, The Newsletter for the Rare Diseases Community.
The production of OrphaNews is realised with the support of Fondation IPSEN, under the aegis of 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
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Editorial Board: Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Anna Bucsics, Ivana Cattaneo, Daria Julkowska, Hélène Dollfus, Alexis Arzimanoglou, Holm Graessner, Henri Jautrou,Julie Bruyere-Zrelli
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