Clinical Research Directory

Inclusion Criteria: * age ≥ 3 years * diagnosis of ALD, as established by elevation of very long chain fatty acid levels or gene mutation * evidence of active cerebral disease as determined by the presence of gadolinium enhancement * ALD MRI (Loes) score ≥ 1 * Patients who have not received prior gene therapy or transplant * Life expectancy of \> 6 months as determined by the enrolling researcher * Have adequate organ function confirmed by laboratory values obtained within 28 days prior to enrollment Exclusion Criteria: * Inability to undergo sedation or MRI studies for any reason * Other concurrent life-threatening disease (life expectancy \<6 months) or eligible for hospice care * Known or suspected hypersensitivity to human serum albumin, diphenhydramine, acetaminophen, methylprednisolone, or any of the components of IMS001.