Novel Therapies for Orphan Diseases-Reference-Cited by-同舟云学术

Novel Therapies for Orphan Diseases

Published:2019-06-18 Issue:7 Volume:10 Page:1020-1023
ISSN:1948-5875
Container-title:ACS Medicinal Chemistry Letters
language:en
Short-container-title:ACS Med. Chem. Lett.

Author:

García Fernández José M.¹^ORCID,Ortiz Mellet Carmen²^ORCID

Affiliation:

1. Instituto de Investigaciones Químicas (IIQ), CSIC—Universidad de Sevilla, Avda. Américo Vespucio 49, Isla de la Cartuja, 41092 Sevilla, Spain

2. Department of Organic Chemistry, Faculty of Chemistry, University of Seville, C/Profesor García González 1, 41012 Seville, Spain

Publisher

American Chemical Society (ACS)

Subject

Organic Chemistry,Drug Discovery,Biochemistry

Link

https://pubs.acs.org/doi/pdf/10.1021/acsmedchemlett.9b00242

Reference12 articles.

1. FDA news release May 24th, 2019: FDA approves innovative gene therapy to treat pediatric patients with spinal muscular atrophy, a rare disease and leading genetic cause of infant mortality. https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-disease.

2. Investigating the landscape of US orphan product approvals

3. Estimating the clinical cost of drug development for orphan versus non-orphan drugs

4. Outrageous prices of orphan drugs: a call for collaboration

5. Drivers of Orphan Drug Development

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