| Article ID | Journal | Published Year | Pages | File Type |
|---|---|---|---|---|
| 8690449 | Pediatric Neurology | 2018 | 49 Pages |
Abstract
These trajectories are not consistent with the typical trajectory of the disease and suggest that intrathecal 2-hydoxypropyl-β-cyclodextrin has stabilized the disease over an extended period of time, supporting the current phase 2/3 controlled registration trial with VTS-270.
Keywords
Related Topics
Life Sciences
Neuroscience
Developmental Neuroscience
Authors
Elizabeth MD, PhD, Jamie BS, Anne PhD, Amy AuD, Robin AuD, Lisa PhD, MPH, Katherine RN, Mariana MD, Daniel S. MD, Forbes D. MD, PhD, Joan A. PhD, PT,