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Best Practices and Standard Protocols as a Tool to Enhance Translation for Neuromuscular Disorders

Willmann, Raffaella and De Luca, Annamaria and Nagaraju, Kanneboyina and Rüegg, Markus A.. (2015) Best Practices and Standard Protocols as a Tool to Enhance Translation for Neuromuscular Disorders. Journal of Neuromuscular Diseases, 2 (2). pp. 113-117.

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Abstract

Recent years witnessed an exciting increase in the number of clinical trials for neuromuscular disorders, in particular for Duchenne Muscular Dystrophy and Spinal Muscle Atrophy. Given the high emotional impact of such developments for devastating diseases with an urgent medical need, it is particularly important to justify human trials on the basis of robust preclinical studies and to avoid a waste of hopes and of funds. This review focuses the discussion on the quality in the conduct clinically-oriented preclinical assessments in rare neuromuscular disease models and on the importance in reporting of preclinical confirmatory studies. Accordingly, it invites scientists, journal publishers and funding agencies to require quality standards to improve translatability of preclinical findings. Recent years witnessed an exciting increase in the number of clinical trials for neuromuscular disorders, in particularfor Duchenne Muscular Dystrophy and Spinal Muscle Atrophy. Given the high emotional impact of such developments fordevastating diseases with an urgent medical need, it is particularly important to justify human trials on the basis of robustpreclinical studies and to avoid a waste of hopes and of funds.This review focuses the discussion on the quality in the conduct clinically-oriented preclinical assessments in rare neuromusculardisease models and on the importance in reporting of preclinical confirmatory studies. Accordingly, it invites scientists,journal publishers and funding agencies to require quality standards to improve translatability of preclinical findings.
Faculties and Departments:05 Faculty of Science > Departement Biozentrum > Neurobiology > Pharmacology/Neurobiology (Rüegg)
UniBasel Contributors:Willmann, Raffaella and Rüegg, Markus A.
Item Type:Article, refereed
Article Subtype:Research Article
Publisher:IOS Press
ISSN:2214-3599
e-ISSN:2214-3602
Note:Publication type according to Uni Basel Research Database: Journal article
Language:English
Identification Number:
Last Modified:02 Oct 2018 13:09
Deposited On:02 Feb 2018 14:14

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