Literature DB >> 32313251

Boosting delivery of rare disease therapies: the IRDiRC Orphan Drug Development Guidebook.

Anneliene Hechtelt Jonker, Virginie Hivert, Michela Gabaldo, Liliana Batista, Daniel O'Connor, Annemieke Aartsma-Rus, Simon Day, Ken Sakushima, Diego Ardigo.   

Abstract

Mesh：

Year:  2020        PMID： 32313251     DOI： 10.1038/d41573-020-00060-w

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


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  3 in total

1.  Orphan Medicine Incentives: How to Address the Unmet Needs of Rare Disease Patients by Optimizing the European Orphan Medicinal Product Landscape Guiding Principles and Policy Proposals by the European Expert Group for Orphan Drug Incentives (OD Expert Group).

Authors:  Annemieke Aartsma-Rus; Marc Dooms; Yann Le Cam
Journal:  Front Pharmacol       Date:  2021-12-16       Impact factor: 5.810

Review 2.  Advances in orphan drug development: Time to change the status and stereotype.

Authors:  Sayanta Thakur
Journal:  Perspect Clin Res       Date:  2022-06-30

Review 3.  Systematic Review: Drug Repositioning for Congenital Disorders of Glycosylation (CDG).

Authors:  Sandra Brasil; Mariateresa Allocca; Salvador C M Magrinho; Inês Santos; Madalena Raposo; Rita Francisco; Carlota Pascoal; Tiago Martins; Paula A Videira; Florbela Pereira; Giuseppina Andreotti; Jaak Jaeken; Kristin A Kantautas; Ethan O Perlstein; Vanessa Dos Reis Ferreira
Journal:  Int J Mol Sci       Date:  2022-08-05       Impact factor: 6.208
  3 in total

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