Clinical trials
on Pelizaeus-Merzbacher Disease
Orbit trial – Interventional – NCT06150716
Evaluating the safety and tolerability of ION356, an antisense oligonucleotide targeting PLP1
Clinicaltrial.gov Ionis websiteRocket trial – Observational – NCT05659901
Study on the natural history of PMD
Clinicaltrial.gov Ionis website