Pelizaeus-Merzbacher disease
RecruitingStudy on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease
Study on the Safety and Effects of ION356 for Patients with Pelizaeus-Merzbacher Disease
- Disease:Pelizaeus-Merzbacher disease
- Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna
The Netherlands- Participants:0–17 years
- Substances:2'-O-(2-Methoxyethyl) Modified Antisense Oligonucleotide Targeting Plp1 Pre-Mrna
- Sponsor:Ionis Pharmaceuticals Inc.















