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Summary of Edition of 15 March 2023

Looking back at Rare Disease Day 2023

Endo-ERN webinar: Getting to know rare disease registries

ERKNet: New patient website

Join EuroBloodNet at the International neurovascular training course on sickle cell disease

TransplantChild: Education and Training query

Australia's National Disability Insurance Scheme: Strategic priorities for improvement

Estimating the costs of rare diseases in Hong Kong

Leveraging policy to support caregivers in Latin America

USA: NORD announces 2023 Rare Impact Award honourees

EMA scientific advice for medical devices pilot: Applications open

EMA Human Medicines Highlights 2022

HOPE states position on the European Health Data Space

Improving orphan drug access in Spain: Strategic discussion and proposals

Why are orphan drugs so expensive? Comparing the costs of FDA-approved medicines

Redistributing resources to curb increasing drug prices

Managing risk in orphan drug reimbursement

Current and future therapeutics for paediatric genetic diseases

EMA: One new positive opinion on Market Authorisation with an Orphan Designation in February 2023

Sixteen new positive opinions for orphan designation accorded in February 2023 by the COMP

Seven new Market Authorisations granted in February 2023 by the FDA

Twenty-three new orphan designations approved in February 2023 by the FDA

What do we know about health emigration in rare diseases?

The role of patient journey tools in improving rare disease care

Global Genes 2023 RARE Health Equity Report: Improving access to care for underserved communities

Away from "one disease at a time": New approaches to screening and drug development

Considerations for expanding reproductive genetic carrier screening: Evidence from Belgium

Quality of life and other social effects on people living with rare skin diseases

Challenges in studying rare diseases of epigenetic origin

RareData EU: Guidance for real world data studies and building registries

Considerations for adopting whole genome sequencing in paediatrics

Incidence and age distribution of rare cancers in Hiroshima Prefecture, Japan

Epidemiological characteristics of rare cancers in South Asia

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

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)

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