International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases-Reference-Cited by-同舟云学术

International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases

Published:2019-04-12 Issue:5 Volume:11 Page:
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Boycott Kym M¹^ORCID,Lau Lilian PL²³,Cutillo Christine M³,Austin Christopher P³

Affiliation:

1. Children's Hospital Eastern Ontario Research Institute University of Ottawa Ottawa ON Canada

2. IRDiRC Scientific Secretariat Paris France

3. National Center for Advancing Translational Sciences (NCATS) National Institutes of Health (NIH) Bethesda MD USA

Publisher

EMBO

Subject

Molecular Medicine

Link

https://onlinelibrary.wiley.com/doi/pdf/10.15252/emmm.201910486

Reference9 articles.

1. Future of Rare Diseases Research 2017-2027: An IRDiRC Perspective

2. Progress in Rare Diseases Research 2010-2016: An IRDiRC Perspective

3. EMA(2018)European Medicines Agency (EMA): orphan medicines figures 2000–2017.https://www.ema.europa.eu/documents/other/orphan-medicines-figures-2000-2017_en.pdf. Accessed 20 November 2018

4. FDA(2018)US Food and Drug Administration (FDA): orphan drug designations and approvals.https://www.accessdata.fda.gov/scripts/opdlisting/oopd/. Accessed 20 November 2018

5. Late diagnosis of cerebral folate deficiency: Fewer seizures with folinic acid in adult siblings

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