Fosigotifator is an investigational drug being researched for the treatment of Vanishing White Matter disease in adult, pediatric and infant participants. This is a 201-week, open-label, multiple cohort study enrolling adults, pediatric and infant participants with Vanishing White Matter disease.
Participants will attend regular visits during the course of the study and complete medical assessments, blood tests, questionnaires, and be evaluated for side effects.
Males and females >= 6 months of age at the time of Screening.
A clinical diagnosis by a physician experienced in the assessment of VWM disease; and
A molecular diagnosis of VWM disease, and
A magnetic resonance imaging (MRI) presentation consistent with VWM disease.
Pediatric participants >= 6 months and < 6 years of age must not be on any form of respiratory support at the time of Screening.
Changes in medication use for the management of VWM disease symptoms within the 4 weeks preceding Screening.
Seizure disorder not considered adequately controlled by the investigator within the 6 months preceding Screening.
Participant who, in the opinion of the investigator, is incapable of completing study-required visits and procedures to assess primary and secondary endpoints.