| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 6041492 | Neuromuscular Disorders | 2015 | 10 Pages |
Abstract
â¢The manuscript elaborates on what is clinically meaningful and how to measure this in DMD.â¢We discuss methods of efficacy measurement - strength versus function.â¢We discuss animal models and biochemical outcome measures for DMD.â¢We review the strategy and design of clinical studies - extrapolating to other stages of the disease.
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Authors
Stephen Lynn, Annemieke Aartsma-Rus, Kate Bushby, Pat Furlong, Nathalie Goemans, Annamaria De Luca, Anna Mayhew, Craig McDonald, Eugenio Mercuri, Francesco Muntoni, Marita Pohlschmidt, Jan Verschuuren, Thomas Voit, Elizabeth Vroom, Dominic J. Wells,