X-linked Adrenoleukodystrophy

Speaker: Dr Caroline Sevin;
When: April 18, 2025;
Time: 6 pm CET (7 pm CEST);

About Topic: This Webinar will focus on X-linked adrenoleukodystrophy (ALD), a genetic disorder affecting the nervous system and the most frequent leukodystrophy in children and males. We will discuss clinical symptoms, underlying molecular mechanisms and therapeutic approaches. We will also highlight the crucial role of early screening for early detection, enabling timely intervention and the importance of continuous follow-up to monitor disease progression and the effectiveness of emerging treatments.

Learning Objective:

• Discuss the different phenotypes of ALD, focusing on cerebral ALD
• Know how to diagnose and follow X-ALD children and adults
• Evaluate the indications and limits for HSCT, gene therapy and new therapeutics
• Recognise the critical importance of early diagnosis and genetic counselling