Study Details

Testing the drug ION356 for males with Pelizaeus-Merzbacher Disease

(IRB#: IRB_00177944)

Pelizaeus-Merzbacher Disease is a disease that causes impairment of the neurons in someone's brain and spinal chord. Neurons are a special type of cell that transmit and receive signals throughout the central nervous system. They send signals to different parts of the central nervous system through the axon of the neuron that is covered in myelin. Myelin helps the signals get from one neuron to another. A protein called PLP1 helps form myelin, and those with PMD have abnormalities with PLP1 that causes little or no myelin to be formed. This makes it difficult for the signals to make it to the next neuron which causes problems in the nervous system. This study wants to see if the drug ION356 will help people with this disease and determine if the medication is safe. Everyone in the study will be treated with the study drug. Being in this study requires up to 22 visits to the study clinic with 3 overnight stays and 31 phone calls over 3 years. Medical tests will be done to track the health of the participants.

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