The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.
Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.
Ionis Pharmaceuticals (IONS) is the sponsoring company for this trial. BiotechSign currently grades this company D (50/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.