Looking back on Rare Disease Day 2024
ReadOrphanet News
Rare Disease Platform welcomes French Minister of Health for Rare Disease Day 2024
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Rare Disease Platform welcomes French Minister of Health for Rare Disease Day 2024
ReadImproving access to gene therapy in LMICs: The hub and spoke model
ReadTowards collaborative approaches to medical device development
ReadEMA: Two positive opinions on market authorisation with an orphan designation in February 2024
ReadEMA: Fourteen new positive opinions for orphan designation accorded by the COMP in February 2024
ReadFDA: Six new market authorisations granted in February 2024
ReadFDA: 41 new orphan designations approved in February 2024
ReadGenetic diagnosis of rare intellectual disability and parental mental health
ReadiSTORE: improving statistical methodologies for rare disease clinical trials
ReadMapping the European registry landscape for rare bone and mineral conditions
ReadMethods for estimating rare disease prevalence
ReadOrphaNews, 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
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Editorial Board: Victoria Hedley, Yann Le Cam, Charlotte Rodwell, Anna Bucsics, Ivana Cattaneo, Daria Julkowska, Alexis Arzimanoglou, Henri Jautrou, Holm Graessner, Julie Bruyere-Zrelli, Andrea Osvoll, Stanislav Ostapenko, Valentina Bottarelli, Dave Pearce, Samantha Parker, Alexandra Heumber Perry
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