Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach-Reference-Cited by-同舟云学术

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Longitudinal natural history studies based on real-world data in rare diseases: Opportunity and a novel approach

Published:2024-05 Issue:1 Volume:142 Page:108453
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Adang Laura Ann,Sevagamoorthy Anjana,Sherbini Omar,Fraser Jamie L.,Bonkowsky Joshua L.,Gavazzi Francesco,D'Aiello Russel,Modesti Nicholson B.,Yu Emily,Mutua Sylvia,Kotes Emma,Shults Justine,Vincent Ariel,Emrick Lisa T.,Keller Stephanie,Van Haren Keith P.,Woidill Sarah,Barcelos Isabella,Pizzino Amy,Schmidt Johanna L.,Eichler Florian,Fatemi Ali,Vanderver Adeline

Funder

National Institutes of Health

NINDS

Publisher

Elsevier BV

Reference70 articles.

1. What is the purpose of the orphan drug act?;Herder;PLoS Med.,2017

2. How many rare diseases are there?;Haendel;Nat. Rev. Drug Discov.,2020

3. Epidemiology of rare diseases is important;Bruckner-Tuderman;J. Eur. Acad. Dermatol. Venereol.,2021

4. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database;Nguengang Wakap;Eur. J. Hum. Genet.,2020

5. A guide for the diagnosis of rare and undiagnosed disease: beyond the exome;Marwaha;Genome Med.,2022

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