| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 5632197 | Neuromuscular Disorders | 2017 | 9 Pages |
Abstract
â¢Increasing number of drug development programmes present opportunities and challenges.â¢Increased focus on rigour and de-risking of both pre-clinical and clinical studies.â¢Focus on lessening burden on patients and families, reducing cost of development.â¢Increased patient engagement in design, execution, and regulatory processes key.â¢TREAT-NMD provides an open discussion for these opportunities and challenges.
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Authors
E.P. Hoffman, Workshop Participants Workshop Participants,