Expert Webinar

FUTURE HORIZONS IN NEURONOPATHIC MPS II:
A QUEST FOR VALID CLINICAL AND BIOCHEMICAL ENDPOINTS

SESSION FOR HEALTHCARE PROFESSIONALS

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Speakers

Joseph Muenzer, MD, PhD

Professor of Pediatrics and Genetics, University of North Carolina at Chapel Hill, Chapel Hill, NC, USA

Elsa Shapiro, PhD

Professor, Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA
Consultant, Shapiro Neuropsychology Consulting.

Maria Fuller, PhD

Professor, Genetics and Molecular Pathology, SA Pathology, Adelaide, South Australia

Program

Welcome and introduction

Professor Joseph Muenzer

The impact of cognitive and behavioral impairment on quality of life in patients with mucopolysaccharidosis II (MPS II)
The need for assessment and monitoring
The emerging role of biomarkers and surrogates

Neuronopathic MPS II: where are we now?

Professor Joseph Muenzer

Introduction to MPS II and overview of neuronopathic disease

Impact of neuronopathic disease on patients and families

Unmet needs in neuronopathic MPS II, including early diagnosis/prediction of cognitive impairment, need for specific therapies that reach the target cells and organelles within the central nervous system (CNS)

Need for monitoring response to treatment; current knowledge/approaches – for example cognitive testing, magnetic resonance imaging, cerebrospinal fluid (CSF) glycosaminoglycans (GAGs).

Challenges in assessing cognition and behavior in patients with MPS II

Professor Elsa Shapiro

Natural history of cognitive/behavioral aspects of MPS II

Selection of cognitive tests suitable for use in patients with MPS II

Challenges associated with cognitive testing in patients with MPS II, including those associated with obtaining an expert cognitive and behavioral assessment

Considerations for interpretation of cognitive test results in MPS II

A role for biomarkers in MPS II

Professor Maria Fuller

Need for biomarkers in neuronopathic MPS II, for both diagnosis/prediction of CNS disease and for patient monitoring

Limitations of current laboratory measures in clinical research (e.g. CSF GAGs, heparan sulfate)

Consideration of CSF disaccharides and GM2 and GM3 gangliosides as potential biomarkers

Future directions, including consideration of use of other sample types

Faculty panel discussion with live Q&A

Prof Joseph Muenzer

Prof Elsa Shapiro

Concluding remarks

Prof Joseph Muenzer

This meeting is initiated, organized and funded by Takeda
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VV-MEDMAT-23775 Date of preparation: September 2020.