EPNS 2019 Congress Industry Sessions

Wednesday, 18 September 2019 Trianti Hall

13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 1
From CLN2 diagnosis to treatment: How to keep improving?

Chair: Maria Mazurkiewicz-Bełdzińska (POLAND)

Welcome & Introduction

Maria Mazurkiewicz-Bełdzińska (POLAND)

Lifting the curtain: Key hallmarks of CLN2 disease

Maria Mazurkiewicz-Bełdzińska (POLAND)

Diagnosis to treatment: Hospital/centre readiness

Igor Prpić (CROATIA)

Striving towards treatment excellence

Hidde H. Huidekoper (NETHERLANDS)

Interactive discussion: advancing care together

All

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 2
Challenges of Developmental Epileptic Encephalopathies: shaping thefuture?

Co-Chairs: Artemis Gika (GREECE), Alexis Arzimanoglou (FRANCE)

13:00-13:10 Welcome & Introduction

                   Artemis Gika (GREECE)

13:10-13:30 Clinical management: where are we today?

                   Alexis Arzimanoglou (FRANCE)

13:30-13:50 Clinical studies: where do we need to be?

                   Jacqueline French (USA)

13:50-14:10 Clinical decisions: where can we make progress?

                   Lieven Lagae (BELGIUM)

14:10-14:30 Panel Discussion

                   All Faculty

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 3
Time is motor neuron in SMA

Chair: Eduardo Tizzano (SPAIN)

13:00-13:05 Welcome and Introduction

                   Eduardo Tizzano (SPAIN)

13:05-13:25 Nusinersen ▼in infants who initiate treatment in a pre-symptomatic stage of SMA:interim results from the phase 2 NURTURE study (March 2019)

                   Enrico Bertini (ITALY)

13:25-13:45 Accelerating time to diagnosis in SMA

                   Ulrike Schara (GERMANY)

13:45-14:05 Prognostic biomarkers in SMA

                   Basil T. Darras (USA)

14:05-14:25 Emerging challenges of the new SMA phenotypes

                   Francesco Muntoni (UNITED KINGDOM)

14:25-14:30 Summary and close

                   Eduardo Tizzano (SPAIN)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 4
Dravet Syndrome from childhood to adulthood: how to make a successful transition?

Chair: Rima Nabbout (FRANCE)

13:00-13:05 Introduction

                   Rima Nabbout (FRANCE)

13:05-13:20 Managing Dravet Syndrome through adolescence and adulthood: the parents’

                   perspective

                   Isabella Brambilla (SWITZERLAND)

13:20-13:45 Comorbidities associated with Dravet Syndrome, evolution and management

                   Tiziana Granata (ITALY)

13:45-14:10 How to handle the therapeutical transition?

                   Stephane Auvin (FRANCE)

14:10-14:25 Questions / Answers

14:25-14:30 Conclusion

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 5
Adolescence is complicated enough: now add Epilepsy

Chair: Argirios Dinopoulos (GREECE)

13:00-13:05 Welcome – Introduction: improving life-chances in Epilepsy

                   Argirios Dinopoulos (GREECE)

13:05-13:30 Challenges of living with Epilepsy in adolescence

                   Georgia Ramantani (SWITZERLAND)

13:30-13:55 Do we under-recognise sleep disturbances in adolescents with Epilepsy?

                   Adrian Garcia Ron (SPAIN)

13:55-14:20 Managing an increased risk for cognitive dysfunction

                   Kimford Meador (USA)

14:20-14:30 Q&A: What does this mean for everyday clinical practice?

                   All Faculty

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18:30-20:00 INDUSTRY SPONSORED SYMPOSIUM 6
Gene Therapy in SCN1A-Related Epilepsies (Dravet Syndrome)

Co-Chairs: Andreas Brunklaus (UNITED KINGDOM), Stephanie Schorge (UNITED KINGDOM)

Faculty: Rubén Hernández (SPAIN), Gabriele Lignani (UNITED KINGDOM), Benit Maru (USA)

18:30-18:45  Overview and background of SCN1A-related epilepsies

18:45-19:00  Introduction to gene therapy

19:00-19:45  Current gene therapy approaches for SCN1A-related epilepsies

19:45-20:00  Q&A session

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18:30-20:00 INDUSTRY SPONSORED SYMPOSIUM 7
Rewriting the tale of Sisyphus: the SMA boulder that went up amountain and came down to change a disease

18:30-19:00 Re-writing my story of SMA – How each patient journey is different

                   Edward Ndopu (SOUTH AFRICA)

19:00-19:20 The SMA patient journey through a clinician’s eyes – What milestones have weachieved

                   Francesco Muntoni (UNITED KINGDOM)

19:20- 19:40 Natural history and revolution of the disease – Do we know what the future willholdfor SMA and how can we prepare for it

                   Eugenio Mercuri (ITALY)

19:40- 20:00      Presenter discussion – Q&A

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18:30-20:00 INDUSTRY SPONSORED SYMPOSIUM 9
Evolution of diagnosis and management in Cerebral Adrenoleukodystrophy

18:30-18:45 Light Dinner Buffet

18:45-18:50 Introductions and Programme Overview

                   Jorn-Sven Kuhl (GERMANY)

18:50-19:10 Clinical presentation of Cerebral Adrenoleukodystrophy: the importance of earlydiagnosis and treatment

                   Isabella Moroni (ITALY)

19:10-19:25 Treatment of Cerebral Adrenoleukodystrophy with allogeneic hematopoietic stemcell transplantation

                   Jorn-Sven Kuhl (GERMANY)

19:25-19:40 Advanced Neuroimaging in the diagnosis and post-treatment monitoring ofCerebral Adrenoleukodystrophy

                   Florian Eichler (USA)

19:40-19:45 Closing Remarks

                   Jorn-Sven Kuhl (GERMANY)

19:45-20:00 Q&A

                   All Faculty

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 11
Duchenne Muscular Dystrophy: from suspicion to treatment

Chair: Eugenio Mercuri (ITALY)

13:00-13:05 Welcome and introduction

                   Eugenio Mercuri (ITALY)

13:05-13:30 The importance of early diagnosis and timely treatment and management forpatients with DMD

                   Gunther Bernert (AUSTRIA)

13:30-13:50 Ataluren for the treatment of patients with nmDMD*: latest developments

                   Eugenio Mercuri (ITALY)

13:50-14:10 Management of nmDMD in patients: clinician’s case study

                   Talia Dor (ISRAEL)

14:10-14:30 Today’s key learnings and Q&A

                   Eugenio Mercuri (ITALY)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 12
Severe Paediatric Hypersalivation: its impact on quality of life and the need to manage it effectively

Chair: Coriene Catsman-Berrevoets (NETHERLANDS)

13:00-13:05 Welcome & Introduction

                   Coriene Catsman-Berrevoets (NETHERLANDS)

13:05-13:30 The Importance of effective assessment and management of Sialorrhea in childrenwith Cerebral Palsy

                   Lindsey Pennington (UNITED KINGDOM)

13:30-13:55 Why it is time to reconsider our approach to managing Hypersalivation

                   Jeremy Parr (UNITED KINGDOM)

13:55-14:20 Are you a tablet crusher?

                   Alastair Patterson (UNITED KINGDOM)

14:20-14:27 Panel Discussion & Interactive Session

                   Coriene Catsman-Berrevoets (NETHERLANDS)

14:27-14:30 Close

                   Coriene Catsman-Berrevoets (NETHERLANDS)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 13
The latest & the future: management of LGS and related Epileptic Encephalopathies

13:00-13:05  Introduction

                   Stephane Auvin (FRANCE)

13:05-13:25 Non-pharmacological treatments for LGS: integration new treatmentrecommendations

                   Stephane Auvin (FRANCE)

13:25-13:55 Beyond seizures: improving cognitive and behavioral outcomes

                   Nicola Specchio (ITALY)

13:55-14:15 The current and advances in pharmacological treatments

                   Sophia Varadkar (UNITED KINGDOM)

14:15-14:30 Closing and Q&A

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 14
Gene therapy in Neuromuscular Disorders: the time is now

Chair: Kevin Strauss (USA)

13:00-13:15 Welcome and Introduction

                   Kevin Strauss (USA)

13:15-13:40 Gene therapy: the past, present and future

                   Arthur Burghes (USA)

13:40-14:05 Gene therapy and Neuromuscular Disorders: the impact on clinical practice

                   Francesco Muntoni (UNITED KINGDOM)

14:05-14:25 Panel discussion

                   All Faculty

14:25-14:30 Symposium close

                   Kevin Strauss (USA)

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13:00-13:30 INDUSTRY SPONSORED SYMPOSIUM 15
Chronic Drooling in children – Consequences and management – A new era

Chair: Roser Pons (GREECE)

13:00-13:05 Introduction

13:05-13:25 Presentation on Chronic Drooling in children – Consequences and management  - Anew era

                   Rajat Gupta (UNITED KINGDOM)

13:25-13:30 Questions

                   Helen Shaw (UNITED KINGDOM)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 16
A sense of urgency and the challenging consequences of early-onset rare Epilepsies

Chair: Lieven Lagae (BELGIUM)

Welcome and introduction

Lieven Lagae (BELGIUM)

A sense of urgency in the treatment of rare Epileptic Encephalopathies

Richard Chin (UNITED KINGDOM)

The social and financial burden of living with rare epilepsies

Adam Strzelczyk (GERMANY)

Pronouncing a diagnosis and coping with the aftermath

Tania Korsak (BELGIUM)

Audience Q&A

All

Closing Remarks

Lieven Lagae (BELGIUM)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 17
Further tales of the unexpected: early diagnosis in childhood muscular disorders

Chair: Maureen Cleary (UNITED KINGDOM)

13:00-13:10 Introduction

                   Maureen Cleary (UNITED KINGDOM)

13:10-13:35 Muscle weakness and hyperCKemia: deciphering the diagnosis

                   Andres Nascimento (SPAIN)

13:35-14:00 Mild motor delay: a common symptom leading to an uncommon diagnosis

                   Federica Deodato (ITALY)

14:00-14:25 Following the clues to successfully diagnose childhood muscle disorders

                   Maureen Cleary (UNITED KINGDOM)

14:25-14:30 Closing remarks

                   Maureen Cleary (UNITED KINGDOM)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 18
Targeting respiratory dysfunction in DMD

Chair: Argirios Dinopoulos (GREECE)

13:00-13:10 Welcome and introduction

                   Argirios Dinopoulos (GREECE)

13:10-13:40 Respiratory function decline as a predictor of clinical outcomes

                   Oscar H. Mayer (USA)

13:40-14:10 Altering the course of respiratory dysfunction in DMD

                   Laurent Servais (FRANCE)

14:10-14:15 Summary

                   Argirios Dinopoulos (GREECE)

14:15-14:30 Q&A and Close

                   All Faculty

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 19
Development of therapies for Genetic Disorders

Diagnostic journey in Neuromuscular Disorders

Alessandra Ferlini (ITALY)

Genetic therapies and application in Neuromuscular Disorders

Eugenio Mercuri (ITALY)

What can we learn from the Nationwide Gene Therapy Experience?

Linda Lowes (USA)

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13:00-14:30 INDUSTRY SPONSORED SYMPOSIUM 20
A Window on something rare: understanding AADC deficiency

Chair: Roser Pons (GREECE)

13:00-13:10 Welcome and Introduction

                   Roser Pons (GREECE)

13:10-13:30 Cerebral palsy mimics and differential diagnosis of AADC deficiency

                   Roser Pons (GREECE)

13:30-13:50 AADC deficiency: clinical diagnosis based on case studies

                   Bruria Ben-Zeev (ISRAEL)

13:50–14:10 Current management options and overview of treatments in development

                   Thomas Opladen (GERMANY)

14:10–14:30 Q&A session

                   Roser Pons (GREECE)

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