The study looks at a rare brain condition called Neurodegeneration with brain iron accumulation. This condition includes subtypes such as pantothenate kinase-associated neurodegeneration and mitochondrial membrane protein-associated neurodegeneration. In this condition, brain cells gradually die (neurodegeneration) and excess iron builds up in the brain, leading to movement problems and loss of function. The medication being tested is an oral capsule containing dimethyl fumarate, which is taken by mouth.
The purpose of the study is to see whether the medication is safe and can slow the worsening of symptoms compared with no treatment. Participants will be randomly assigned to receive either the medication or no additional therapy, and neither the participants nor the study staff will know which group each person is in (blinded). Over about 25 weeks, participants will have regular visits where doctors will check for side effects and will use simple questionnaires, such as the MPAN Disease Rating Scale or the PKAN Disease Rating Scale, to see if the disease is staying stable. Blood samples will also be taken to measure substances that indicate brain injury or inflammation, called biomarkers, which are measured in the blood (serum). After the initial period, participants may continue in an open label extension where everyone receives the medication.
Poland 
