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Translational medicine in neuromuscular disorders: from academia to industry
Although around half of US Food and Drug Administration (FDA)-approved drugs originate from discoveries made in academic research laboratories, the US National Institutes of Health (NIH) estimates that nearly 90% of therapies developed in preclinical stages never reach clinical trials. From those in...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The Company of Biologists Ltd
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6906629/ https://www.ncbi.nlm.nih.gov/pubmed/31658990 http://dx.doi.org/10.1242/dmm.041434 |
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author | Cowling, Belinda S. Thielemans, Leen |
author_facet | Cowling, Belinda S. Thielemans, Leen |
author_sort | Cowling, Belinda S. |
collection | PubMed |
description | Although around half of US Food and Drug Administration (FDA)-approved drugs originate from discoveries made in academic research laboratories, the US National Institutes of Health (NIH) estimates that nearly 90% of therapies developed in preclinical stages never reach clinical trials. From those in clinical trials, only 10% obtain marketing approval. Despite the recent advances in our understanding and diagnosis of neuromuscular disease, and the development of rational therapies in clinical trials, these numbers have not changed dramatically over the past two decades. This article discusses the advantages and challenges for translational research initiated from academia, and the trend towards bridging the gap between discovery and translation to the clinic. A focus is made on recent advances in therapeutic development for neuromuscular disorders. |
format | Online Article Text |
id | pubmed-6906629 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | The Company of Biologists Ltd |
record_format | MEDLINE/PubMed |
spelling | pubmed-69066292020-01-14 Translational medicine in neuromuscular disorders: from academia to industry Cowling, Belinda S. Thielemans, Leen Dis Model Mech Special Article Although around half of US Food and Drug Administration (FDA)-approved drugs originate from discoveries made in academic research laboratories, the US National Institutes of Health (NIH) estimates that nearly 90% of therapies developed in preclinical stages never reach clinical trials. From those in clinical trials, only 10% obtain marketing approval. Despite the recent advances in our understanding and diagnosis of neuromuscular disease, and the development of rational therapies in clinical trials, these numbers have not changed dramatically over the past two decades. This article discusses the advantages and challenges for translational research initiated from academia, and the trend towards bridging the gap between discovery and translation to the clinic. A focus is made on recent advances in therapeutic development for neuromuscular disorders. The Company of Biologists Ltd 2019-10-24 /pmc/articles/PMC6906629/ /pubmed/31658990 http://dx.doi.org/10.1242/dmm.041434 Text en © 2019. Published by The Company of Biologists Ltd http://creativecommons.org/licenses/by/4.0This is an Open Access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0), which permits unrestricted use, distribution and reproduction in any medium provided that the original work is properly attributed. |
spellingShingle | Special Article Cowling, Belinda S. Thielemans, Leen Translational medicine in neuromuscular disorders: from academia to industry |
title | Translational medicine in neuromuscular disorders: from academia to industry |
title_full | Translational medicine in neuromuscular disorders: from academia to industry |
title_fullStr | Translational medicine in neuromuscular disorders: from academia to industry |
title_full_unstemmed | Translational medicine in neuromuscular disorders: from academia to industry |
title_short | Translational medicine in neuromuscular disorders: from academia to industry |
title_sort | translational medicine in neuromuscular disorders: from academia to industry |
topic | Special Article |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6906629/ https://www.ncbi.nlm.nih.gov/pubmed/31658990 http://dx.doi.org/10.1242/dmm.041434 |
work_keys_str_mv | AT cowlingbelindas translationalmedicineinneuromusculardisordersfromacademiatoindustry AT thielemansleen translationalmedicineinneuromusculardisordersfromacademiatoindustry |