Research.Patient.info
Pelizaeus-Merzbacher Disease research hub
2 indexed studies · 2 currently recruiting
- recruiting
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
This study, called 'Rocket', is for boys aged 6 months to 17 years with a rare condition called Pelizaeus-Merzbacher Disease (PMD). It aims to better understand how PMD develops and how it affects the body over time, which could help develop new treatments.
United States · France · Germany - recruitingPHASE1
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
This study is looking at a new treatment called ION356 for boys aged 2-17 with Pelizaeus-Merzbacher Disease (PMD). It aims to see how safe the treatment is and how the boy's body handles it. It's an early-stage study to understand more about this potential new medicine.
United States · France · Germany