Multidisciplinary Management and Treatment in CLN2 (Batten Disease)

This webinar addresses the practical management of CLN2 disease, a rare and rapidly progressive neurodegenerative condition where early diagnosis directly influences treatment opportunities and long-term care planning. Led by Dr Dipak Ram, the session focuses on current treatment approaches, including enzyme replacement therapy, alongside supportive and symptomatic care across the disease trajectory. The discussion emphasizes the importance of coordinated multidisciplinary care, highlighting how early intervention, structured communication, and integrated care models can improve clinical decision-making and reduce burden on families. Participants will gain practical insight into how CLN2 is managed in real-world settings and how earlier recognition translates into more effective long-term outcomes.

The session examines the management of CLN2 disease, focusing on how early diagnosis, treatment, and multidisciplinary coordination intersect to shape patient outcomes.

Expert presentations cover:

• ‍Clinical framing and care planning (Dr Dipak Ram): linking early diagnosis to meaningful treatment and long-term care‍
• Early diagnosis and timing: understanding disease progression and the consequences of delay‍
• Management in practice: treatment approaches, enzyme replacement therapy, and supportive care‍
• Multidisciplinary coordination: integrating MDT input and supporting families across the disease course

The discussion features:

• The impact of delayed diagnosis on treatment opportunities and care planning
• Practical approaches to integrating multidisciplinary care across specialties
• Communication and coordination challenges in real-world clinical settings
• Strategies to reduce fragmentation and improve continuity of care for families

References: (1) Schulz A et al. N Engl J Med 2018;378:1898–1907 (2) Schulz A et al. Lancet Neurol 2024 (3) Mole SE et al. Orphanet J Rare Dis 2021;16:185 (4) Cerliponase alfa. Summary of Product Characteristics. BioMarin International Limited. 12 December 2023; Available at: https://www.ema.europa.eu/en/documents/productinformation/brineura-epar-product-information_en.pdf (5) Cerliponase alfa. Prescribing Information. BioMarin Pharmaceutical Inc. 2024

This webinar is intended for healthcare professionals only.
This is a promotional webinar sponsored and funded by BioMarin. BioMarin medicines will be discussed.

All adverse event(s) discussed will be notified to the Pharmacovigilance Department of BioMarin. BioMarin does not recommend the use of any product in any manner other than that described in the approved Prescribing Information. Prescribing information and adverse event reporting information can be found here.  Regulatory status and approved indications of any product may vary between countries, please check the registration status of any product in your respective country. Please refer to your local Prescribing Information/SmPC before prescribing.