Guanabenz Clinical Trial Data for Vanishing White Matter Patients

This dataset comprises clinical trial data from 33 patients with early childhood onset Vanishing White Matter (VWM) treated with Guanabenz. It was authored by Marjo van der Knaap and last updated in February 2026.

Use Cases

• Analyze safety and tolerability outcomes across the 33-patient cohort to assess Guanabenz's profile in VWM.
• Model the pharmacokinetic profile of Guanabenz using patient-specific dosage and biomarker data.
• Evaluate potential efficacy signals by correlating treatment duration with clinical outcome measures for VWM.
• Investigate patient demographics and baseline characteristics to understand the trial population structure.

Strengths

• Data originates from a defined clinical trial with 33 enrolled patients.
• Focuses on a rare neurological disorder (Vanishing White Matter), providing specific research data.
• Dataset is recent, with a last update recorded in February 2026.

Limitations

• Small sample size of 33 patients limits statistical power for subgroup analyses.
• Specific data columns, file formats, and sample data are unavailable for review.
• The dataset's license terms are unknown, which may restrict usage.

Provenance

Source

DataverseNL Harvested Dataverse

Collection Method

Clinical trial data collection.

Freshness

Last updated 2026-02-23.