ADRENOLEUKODYSTROPHY

Course overview

ADRENOLEUKODYSTROPHY IS A PEROXISOMAL DISORDER RESULTING FROM ABNORMAL METABOLISM OF THE VERY-LONG-CHAIN FATTY ACIDS (VLCFA). IT IS CLASSIFIED INTO DIFFERENT SUBTYPES BASED ON THE MODE OF INHERITANCE, CLINICAL PRESENTATION, AGE OF ONSET, AND ORGANS INVOLVED. THE PROGNOSIS IS POOR, AND THE MAJORITY OF AFFECTED PATIENTS WILL DEVELOP NEUROLOGICAL DISABILITIES AND DEATH. THIS ACTIVITY REVIEWS THE PATHOPHYSIOLOGY, EVALUATION, MANAGEMENT, AND PROGNOSIS OF THE DIFFERENT ADRENOLEUKODYSTROPHY TYPES AND HIGHLIGHTS THE NEW UPDATES ABOUT THE DIAGNOSIS AND TREATMENT OPTIONS THAT ARE AVAILABLE OR UNDER INVESTIGATION FOR THIS CONDITION.