Update on Standard Operating Procedures in Preclinical Research for DMD and SMA Report of TREAT-NMD Alliance Workshop, Schiphol Airport, 26 April 2015, The Netherlands

Maaike van Putten; Annemieke Aartsma-Rus; Miranda D Grounds; Joe N Kornegay; Anna Mayhew; Thomas H Gillingwater; Shin'ichi Takeda; Markus A Rüegg; Annamaria De Luca; Kanneboyina Nagaraju; Raffaella Willmann

doi:10.3233/JND-170288

Update on Standard Operating Procedures in Preclinical Research for DMD and SMA Report of TREAT-NMD Alliance Workshop, Schiphol Airport, 26 April 2015, The Netherlands

J Neuromuscul Dis. 2018;5(1):29-34. doi: 10.3233/JND-170288.

Authors

Affiliations

¹ Department of Human Genetics, Leiden University Medical Center, Leiden, The Netherlands.
² School of Anatomy, Physiology and Human Biology, The University of Western Australia, Perth, WA, Australia.
³ College of Veterinary Medicine and Biomedical Sciences, Texas A&M University, TX, USA.
⁴ John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle, UK.
⁵ Edinburgh Medical School, Biomedical Sciences, University of Edinburgh, Edinburgh, UK.
⁶ National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan.
⁷ Biozentrum, University of Basel, Basel, Switzerland.
⁸ Department of Pharmacy and Drug Sciences, Unit of Pharmacology, University of Bari, Bari, Italy.
⁹ School of Pharmacy and Pharmaceutical Sciences, Binghamton University, NY, USA.
¹⁰ Swiss Foundation for Research on Muscle Diseases, Cortaillod, Switzerland.

Abstract

A workshop took place in 2015 to follow up TREAT-NMD activities dedicated to improving quality in the preclinical phase of drug development for neuromuscular diseases. In particular, this workshop adressed necessary future steps regarding common standard experimental protocols and the issue of improving the translatability of preclinical efficacy studies.

Keywords: DMD; SMA; preclinical; standard operating procedures; translational research.

Publication types

Congress

MeSH terms

Animals
Biomedical Research
Disease Models, Animal
Drug Discovery
Education
Humans
Muscular Atrophy, Spinal / drug therapy*
Muscular Dystrophy, Duchenne / drug therapy*
Netherlands
Outcome Assessment, Health Care
Quality Improvement*
Translational Research, Biomedical*