Targeting shared molecular etiologies to accelerate drug development for rare diseases

Targeting shared molecular etiologies to accelerate drug development for rare diseases

Abstract Rare diseases affect over 400 million people worldwide and less than 5% of rare diseases have an approved treatment. Fortunately, the number of underlying disease etiologies is far less than the number of diseases, because many rare diseases share a common molecular etiology. Moreover, many...

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Main Authors: Galliano Zanello, Macarena Garrido‐Estepa, Ana Crespo, Daniel O'Connor, Rima Nabbout, Christina Waters, Anthony Hall, Maurizio Taglialatela, Chun‐Hung Chan, David A Pearce, Marc Dooms, Philip John Brooks
Format: Article
Language:English
Published: Springer Nature 2023-06-01
Series:EMBO Molecular Medicine
Subjects:
basket clinical trials
IRDiRC
rare diseases
shared molecular etiologies
therapeutic development
Online Access:https://doi.org/10.15252/emmm.202217159
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https://doi.org/10.15252/emmm.202217159

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