Excellence in Pediatrics Institute
Think Rare Program
Think Rare Program  ·  CLN2 (Batten Disease) September 2026

This Issue

New Live Webinar:
Multidisciplinary Management and Treatment in CLN2 (Batten Disease)

The Think Rare Program continues with a live webinar on the management and treatment of CLN2 (Batten disease), led by Dr. Dipak Ram, Consultant Pediatric Neurologist at Royal Manchester Children’s Hospital.

Context

Managing CLN2 means building a service around the child and family. Dr. Ram’s clinical experience explores how a dedicated multidisciplinary team can transform ongoing care.

The initial diagnosis is an important point in the care journey. Giving families clear information about CLN2, treatment options, and the long-term outlook can help them understand what to expect and how care will be managed.

This webinar, taking place on Wednesday, 9 September and presented by Dr. Dipak Ram, draws on real-world case experience to show how monitoring clinical markers of progression, carefully exploring apparent changes in a patient’s condition, and keeping families informed can shape day-to-day decision-making.

Upcoming Webinar 3 • Wednesday, 9 September

Wednesday, 9th September 2026
13:00 Central European Summer Time (CEST)

Dr. Dipak Ram, Clinical Lead for the CLN2 Service, Royal Manchester Children’s Hospital

Multidisciplinary Management and Treatment in CLN2 (Batten Disease)

A 45-minute webinar on the practical, multidisciplinary management of CLN2 disease. Dr. Dipak Ram shares first-hand experience of setting up and running a specialist Batten disease service, from structuring the multidisciplinary team to delivering a diagnosis and supporting families afterward. The session also covers how to track clinical markers of progression, drawing on real case examples to illustrate the decisions clinicians face as the disease advances.

Register for Webinar 3 →

Key Learning Points from the Webinar

At the end of the webinar, participants will be able to:

1

Building a Multidisciplinary Service

Describe how a dedicated multidisciplinary team and structured “one-stop” specialist clinics can support the ongoing management of CLN2 disease.

2

Delivering the Diagnosis

Apply a structured approach to delivering a CLN2 diagnosis, covering the family’s questions, therapeutic implications, and long-term outlook.

3

Tracking Progression and Making Decisions

Recognize clinical markers of disease progression and apply real-world case lessons to inform decisions about treatment and quality of life.

Did You Miss the Previous Sessions in the Series?Catch Up • Webinar 2

Distinguishing CLN2: Navigating the Differential Diagnosis and Referral Pathways

Distinguishing CLN2: Navigating the Differential Diagnosis and Referral Pathways

Wednesday, 15th July 2026 · 13:00 CEST

Missed the live webinar? You can now watch it on demand. A 60-minute expert-led session with Dr. Marina Trivisano and Prof. Pasquale Striano on distinguishing CLN2 disease from more common pediatric neurological, developmental, and behavioral presentations, covering reassessment frameworks, escalation, and referral pathways.

Watch Webinar 2 On Demand →

Catch Up • Webinar 1

Wednesday, 10th June 2026
16:30 Central European Summer Time (CEST)

Recognizing the Early Signs of CLN2 (Batten Disease)

Missed the live webinar? You can now watch it on demand. A 45-minute expert-led webinar on recognizing the early signs of CLN2 disease. The session helps clinicians identify early symptom patterns, recognize disease progression and clustering cues, and understand when concern should trigger escalation, referral, and genetic testing.

Watch Webinar 1 On Demand →

Disclaimer

These webinars are intended for healthcare professionals only. These are promotional webinars 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. Regulatory status and approved indications of any product may vary between countries; please check the registration status of any product in your respective country.

About the Program

The EiP Think Rare Program empowers pediatricians and general practitioners to identify rare and underdiagnosed conditions early. The program currently covers MPS, MLD, AM, and CLN2 (Batten disease), forming a practical, centralized resource for recognizing rare diseases in clinical practice.

Explore the Learning Zones

Each Learning Zone focuses on a specific rare disease, equipping you with condition-specific knowledge, early signs to watch for, and up-to-date clinical resources. Explore all zones to build both depth in individual conditions and a broader understanding of how rare diseases present in practice.

Mucopolysaccharidosis (MPS)

Explore practical resources to help detect early signs, reduce misdiagnosis, and ensure timely referrals and intervention.

Visit Zone →

Metachromatic Leukodystrophy (MLD)

Recognize early signs often mistaken for common conditions and take action sooner to change the course of a child’s life.

Visit Zone →

Alpha-Mannosidosis (AM)

Recognize the early signs and transform outcomes with earlier, more accurate referral and patient care.

Visit Zone →

Neuronal Ceroid Lipofuscinosis (CLN2)

Explore practical resources on CLN2 (Batten disease), from early recognition to multidisciplinary management and treatment.

Visit Zone →
BioMarin

The THINK RARE program is independently developed and managed by EiP, including content, topics, and appointed speakers. BioMarin has sponsored and funded EiP’s webinars on CLN2.

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