Preclinical alternative drug discovery programs for monogenic rare diseases. Should small molecules or gene therapy be used? The case of hereditary spastic paraplegias-Reference-Cited by-同舟云学术

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Preclinical alternative drug discovery programs for monogenic rare diseases. Should small molecules or gene therapy be used? The case of hereditary spastic paraplegias

Published:2024-10 Issue:10 Volume:29 Page:104138
ISSN:1359-6446
Container-title:Drug Discovery Today
language:en
Short-container-title:Drug Discovery Today

Author:

Sebastiano Matteo Rossi,Hadano Shinji,Cesca Fabrizia,Caron Giulia,Lamacchia Lorenzo,Francisco Serena,Brusco Alfredo,Pullano Verdiana,Baj Gabriele,Gurgone Antonia,Chiantia Giuseppe,Giustetto Maurizio,Russo Silvia,Higgins Anna J.,van Putte Wouter,Vecchia Stefania Della,Battini Roberta,Maria Santorelli Filippo,Ermondi Giuseppe

Publisher

Elsevier BV

Reference106 articles.

1. Valuing the “burden” and impact of rare diseases: a scoping review;Delaye;Front Pharmacol,2022

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

3. Rare and undiagnosed diseases: from disease-causing gene identification to mechanism elucidation;Wang;Fundam Res,2002

4. Drug repositioning trends in rare and intractable diseases;Sakate;Drug Discov Today,2022

5. Knowledge-based approaches to drug discovery for rare diseases;Alves;Drug Discov Today,2022

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