Research.Patient.info
Cree Leukoencephalopathy research hub
2 indexed studies · 0 currently recruiting
- active not recruitingEARLY_PHASE1
Research Study for Single-Patient Treatment of Cree Leukoencephalopathy/Vanishing White Matter Disease
This study is looking at a new medicine called FGT for a very rare, serious brain condition called Cree Leukoencephalopathy (CLE), which is similar to Vanishing White Matter disease. It aims to see if FGT can help slow down the disease and improve life for a patient who currently has no other treatment options.
Canada - ongoing, recruitingPhase I and Phase II (Integrated)- Other
A Study to Explore the Safety, Tolerability, Pharmacokinetic Profile, and Potential Efficacy of Guanabenz in Patients With Early-Childhood Onset Vanishing White Matter (VWM)
This study is looking at a new medicine called Guanabenz for children with a rare brain condition called Vanishing White Matter (VWM). Researchers want to see if it's safe, how the body handles it, and if it could help treat the condition.
Netherlands