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Rare Disease and Orphan Drugs Journal

C O NT E NT S

Topic: A Landscape Map of the Key Global Rare Disease Organisations

Editorial

1 Special issue on Fabry disease - book 1: editorial
Derralynn Hughes, Guillem Pintos-Morell

2 A landscape map of the key global rare disease organizations
Anneliene Hechtelt Jonker, Daniel J. O’Connor

Perspective

3 Framing the European Rare Diseases field through a structured movement of patient organisations
Maria Cavaller-Bellaubi, Virginie Hivert, Roseline Favresse, Yann Le Cam

4 The European joint programme on rare diseases: building the rare diseases research ecosystem
Yanis Mimouni, Juliane Halftermeyer, Yanna Petton, Pauline Adam, Clément Moreau, Ana Rath,
Roseline Favresse, Birute Tumiene, Daria Julkowska

5 The division of rare diseases research innovation at the national center for advancing translational sciences,
NIH: mission, history, and current research activities
P. J. Brooks, Alice Chen Grady, Stephen Groft, Linda Ho, Joanne Lumsden, Meera Shah, Eric Sid, Yanji Xu,
Ainslie Tisdale, Jim Dickens, Dominique Pichard, Tiina Urv

6 Paradox of possibilities: the rare landscape in the Asia Pacific Region
Ritu Jain, Chiuhui Mary Wang, Lisa Foster, Eileen Yangyang Li, Yukiko Nishimura, Kin Ping Tsang

7 ERCAL, a regional initiative for rare diseases in Latin America and the Caribbean
Claudia Gonzaga-Jauregui, Carlos Salazar, Jennifer MacDonald, Juergen K.V. Reichardt, Stephen C. Groft

Review

8 The international rare disease research consortium (IRDiRC): making rare disease research efforts more
efficient and collaborative around the world
Chun-Hung Chan, Samantha Parker, David A. Pearce

Rare Disease and Orphan Drugs Journal III