Physician- and Patient-Reported Outcomes in {chi}-linked Adrenoleukodystrophy: Accelerated Roadmap from Patients Insights to Clinical Trials (S46.007)

Conclusions:The aggregation of physician- and patient-reported outcomes in ALD/AMN presents a tremendous opportunity to better understand what matters to patients and to leverage their insights in combination with natural history data for clinical trial design optimization and moving the disease into clinical trial readiness state.Study Supported by:ALD Connect and ALD/AMN patients and their familiesDisclosure: Dr. Sherman has nothing to disclose. Dr. Connors has nothing to disclose. Dr. Walsh has nothing to disclose. Dr. Eichler has received research support from Bluebird Bio and Vertex Pharmaceuticals.

Source: Neurology - April 17, 2017 Category: Neurology Authors: Sherman, A., Connors, E., Walsh, K., Eichler, F. Tags: Child Neurology: Molecular Biology to Clinical Trials Source Type: research