Industry-Supported Symposia

Join our expert faculty in an interactive, peer-to-peer discussion treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Through the use of real-world clinical case studies, the challenges patients face in transitioning from paediatric to adult care will also be discussed, alongside the key outcomes for these patients.

Speakers:

• Luca Bello
  Assistant Professor of Neurology, Department of Neurosciences DNS, University of Padova, Italy

• Ros Quinlivan
  MRC Centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery, London, UK

For more information, click here.

Symposium Objectives:

• Provide an overview of the Gene Therapy landscape and examine current unmet needs for all SMA patient populations
• Demonstrate our evolving understanding on presymptomatic patients with SMA in the context of real-world evidence
• Discuss real-world evidence of the safety and efficacy of gene therapy, including patients with SMA beyond those studied in clinical trials

Agenda & Speakers:

• 13:00 – 13:05 – Welcome & Introduction
  Dr. Sandra P. Reyna (Chair) – VP, Global Medical Affairs and Head of Therapeutic Area SMA, Novartis Gene Therapies, USA

• 13:05 – 13:15 – Gene Therapy Now: Current Landscape and Durable Treatment Responses
  – Present an overview of current gene therapy landscape
  – Document improved clinical outcomes following gene therapy treatment using biodistribution and clinical trial data
  – Demonstrate durability of treatment response by reviewing clinical trial data and long-term follow-up data
  Prof. Dr. Liesbeth De Waele – Pediatric Neurologist, University Hospitals Leuven and Head of Neuromuscular Reference Centre (NMRC) for Children, Leuven, Belgium

• 13:15 – 13:30 – Newborn Screening or Presymptomatic Patients? Adapting The Nosology to Tailor Individualized Prognosis
  – Introduce the proposed nosology for “presymptomatic” patients with SMA
  -Understand the difference of trajectory of patients following this nosology, and the specific challenges
  -Provide clues to manage parent expectations
  Prof. Laurent Servais – Professor of Pediatric Neuromuscular Diseases, MDUK Oxford Neuromuscular Centre, Oxford, United Kingdom and Professor of Child Neurology University and University Hospital of Liège, Belgium

• 13:30 – 13:50 – New Era of SMA Management: RWE and Clinically Meaningful Treatment Outcomes
  – Present RWE to demonstrate safety and efficacy of gene therapy in SMA, including RWE in patients with SMA beyond those in clinical trials
  – Discuss clinically meaningful outcome measures, such as bulbar function outcomes, including speech, swallow and respiratory function, following treatment
  Dr. Andreas Ziegler – Medical Doctor and Consultant for Pediatric Neurology, Department of Neuropediatrics and Metabolic Medicine, Centre for Childhood and Adolescent Medicine, University Hospital Heidelberg, Germany

• 13:50 – 14:00 – Q&A and Closing Remarks
  Dr. Sandra P. Reyna (Chair)

 For more information, click here.

Description:
Explore the role of the dystrophin-associated protein complex in neuromuscular diseases and the development of gene transfer therapies in the management of Duchenne and limb-girdle muscular dystrophy with leading experts.

Chair:

Volker Straub, MD, PhD
Director, Dubowitz Neuromuscular Centre Co-Director, MRC Centre for Neuromuscular Diseases
UCL Great Ormond Street Institute of Child Health & Great Ormond Street Hospital for Children
NHS Foundation Trust
London, UK

 Speakers:

• Eugenio Maria Mercuri, MD, PhD
  Professor of Paediatric Neurology
  Head of the Paediatric Neurology and Psychiatry Unit
  Gemelli Hospital
  Catholic University Foundation
  Rome, Italy
• Craig Zaidman, MD
  Professor of Neurology and Paediatrics Washington University
  Missouri, United States

For more information, click here.

Agenda & Speakers:

• 18:00 – 18:15
  Proposed mechanisms of intrahepatic cholestasis in XLMTM: lessons from animal models
  Emanuela Pannia, PhD, The Hospital for Sick Children, Toronto, Ontario, Canada

• 18:15 – 18:25
  Case studies of intrahepatic cholestasis as part of the natural history of XLMTM
  Andrés Nascimento, MD, Hospital Universitari Sant Joan de Déu, Barcelona, Spain

• 18:25 – 18:40
  Safety update for ASPIRO study
  Michael Lawlor, MD, PhD, Medical College of Wisconsin, Milwaukee, Wisconsin, USA

• 18:40 – 19:00
  Q&A and discussion
  Moderated by Michael Lawlor, MD, PhD

For more information, click here.

Description:
Can we refer patients with myotonic disorders for specialist care more quickly, and are we able to provide them with a more accurate diagnosis than has previously been possible?

Discover how prompt, accurate, disease recognition can help patients with myotonic disorders to understand (and reduce the impact of) their symptoms.

Agenda & Speakers

• Recognising myotonic disorders
  Chair: Prof. Dr Kristl Claeys, University Hospitals Leuven, Belgium

• DM Scope Registry: Classifying myotonic disorders to improve diagnosis
  Dr Guillaume Bassez, CHU Pitié Salpetrière, Assistance Publique Hôpitaux de Paris, France
• Why don’t we meet patients sooner?
  Prof. John Vissing, Rigshospitalet and Copenhagen University, Denmark

• Concluding comments
  Dr Kristl Claeys

For more information, click here.

Description:
Join Profs. Pushpa Narayanaswami, James F. Howard, and Heinz Wiendl discuss the need for different targeted therapies in patients with generalised Myasthenia Gravis (gMG), and how these therapies could impact future therapeutic management.

The symposium will focus on improving clinicians’:

• Knowledge of the factors that contribute to disease progression in gMG
• Ability to interpret emerging data for emerging therapies in the management of uncontrolled gMG and potential impact on practice
• Ability to individualise care of patients with gMG

Agenda & Speakers:

• Welcome and Introduction
  Pushpa Narayanaswami, MD, FAAN

• Pathogenic Contributions in gMG: Why More Than One Therapeutic Approach is Needed
  Heinz Wiendl, MD

• Emerging Targeted Therapies for Uncontrolled gMG: A Tale of Two Targets
  James F. Howard Jr., MD

• With the Patient at the Forefront, How Could Standard of Care in gMG Evolve in the Future?
  Entire Panel

• “Ask the Faculty” and Take-Home Messages
  Entire Panel

For more information, click here.

Description:
The argenx-sponsored satellite symposium ‘From traditional to targeted: Innovations in the changing gMG landscape’ will delve into the current and emerging treatment landscape in gMG. The panel of experts will explore the significant impact on patients’ lives of this rare neuromuscular disorder and the unmet needs associated with current therapies. They will also discuss how innovations could shift therapeutic strategies towards targeted, individualized treatment.

Agenda & Speakers:

• Welcome and introduction, and patient testimonial video
  Prof. Jan De Bleecker

• Today’s gMG treatment reality: Time for a change?
  Prof. Benedikt Schoser

• The emerging treatment landscape: Hope for tomorrow
  Prof. M Isabel Leite

• In conversation: Navigating the changing landscape in gMG
  All speakers

Description:
Join us at the Sanofi sponsored symposium “Pompe Disease – How Could We Achieve Better Outcomes for Patients?” at 13:00pm (CEST), Silver Hall of the Square Convention Centre. Experts Prof. Mark Roberts, Prof. Jordi Díaz Manera, and Prof. Pascal Laforêt will speak on the importance of early and ongoing patient management to prevent Pompe disease progression, and clinically meaningful changes in patient outcomes.

Agenda & Speakers:

• Introduction
  Mark Roberts

• Demonstrating importance of early treatment in Pompe disease: MRI data
  Jordi Díaz-Manera

• Consequences of treatment interruption: Effects of the pandemic
  Pascal Laforêt

• Clinically meaningful changes in outcomes
  Mark Roberts

• Closing and Q&A
  Mark Roberts, Jordi Díaz-Manera, Pascal Laforêt

For more information, click here.

Symposium objectives:

• To highlight the real-world burden of SMA across all types and ages
• To understand the current standard of care in SMA, including an overview of the treatment landscape and available data in children, adolescents, and adults with Type 1, 2 or 3 SMA
• To share best practices for the management of individuals with SMA from birth to adulthood, and to discuss how its implementation may reduce some of the remaining burden for these individuals

Agenda:

• Welcome and introductions
  Prof Laurent Servais (Chair)

• The broad spectrum of individuals with SMA
  Prof Laurent Servais

• Real-world burden of all types of SMA
  All faculty

• Q&A
  All faculty

• Summary and close
  Prof Laurent Servais

Speakers:

• Professor Laurent Servais | MDUK Oxford Neuromuscular Centre, Oxford, UK (Chair)
• Professor Liesbeth De Waele | University Hospitals Leuven, KU Leuven, Leuven, Belgium
• Professor Maggie Walter | Ludwig-Maximilians-University, Munich, Germany

For more information, click here.

Description:
Intravenous immunoglobulins (IVIg) have proven their efficacy and are considered as a first-line therapy in treating patients with chronic inflammatory demyelinating polyneuropathy (CIDP). Efficacy of IVIg is around 80% of all treated patients. For those who don’t respond, different treatment options are possible. In this debate, we will compare two different approaches: aggressive and conservative approaches and discuss the optimal time to consider the response of CIDP patients to IVIg.

Speakers:

• Chairman: Hans-Peter Hartung – Germany
• Pros: Luis Querol – Spain
• Cons: Helmar Lehmann – Germany

Description:
The chronic and fluctuating nature of generalized myasthenia gravis (gMG) has a substantial impact on a patient’s daily life, including their physical, emotional, social, and economic wellbeing. This symposium will provide the opportunity to engage in discussion with our expert faculty, who will be considering the burden of this chronic disease and illustrating how terminal complement inhibition, which targets a primary driver of AChR Ab+ gMG, can improve patient outcomes.

Agenda & Speakers: 

• Chair:  Dr. Pushpa Narayanaswami 
• Managing gMG as a chronic disease – Prof. Andreas Meisel 
• Complement inhibition: Targeting the primary driver of AChR Ab+ gMG – Prof. Nico Melzer 
• Everyday experience with using a complement inhibitor – Dr. Pushpa Narayanaswami 
• Panel Discussion – All speakers 

For more information, click here.

Description:
This symposium is focused on the importance and utility of appropriate outcomes measures in clinical trials to assess the progression of FSHD, a relentlessly progressive disease leading to significant disability and impact on quality of life.  Reachable Workspace (RWS) is a valid and reliable clinical outcome measure of function whereby there has been a historical lack of tools to evaluate upper extremity function in patients with FSHD.  RWS focuses on shoulder and proximal arm function (among the muscle groups most affected by FSHD), correlates with quality-of-life measures, and is sensitive to change in disease progression.  RWS demonstrated nominally statistically significant and clinically meaningful benefit to muscle function in ReDUX4, a Phase 2 double blind placebo-controlled trial of losmapimod, a small molecule selective p38 MAP kinase inhibitor being developed by Fulcrum Therapeutics as the first disease modifying therapy for FSHD. Results from ReDUX4 and other clinical trials informed the study design of REACH, the first Phase 3 double blind placebo-controlled clinical trial in FSHD.

Speakers:

• Olga Mitelman, MD
  Senior Vice President, Medical Affairs
  Fulcrum Therapeutics, Inc.
  United States

• Hanns Lochmüller, MD, PhD, FAAN
  Professor of Neurology
  University of Ottawa
  Canada

• Sabrina Sacconi, MD, PhD
  Professor of Neurology
  Nice University Hospital
  France

For more information, click here.

Description:
Can you recognise the signs of chronic inflammatory demyelinating polyradiculoneuropathy (CIDP)? Meet our patients and put your diagnostic skills to the test as we navigate through the maze of mimics.

Agenda & Speakers:

• Welcome, introductions and objectives
  Claudia Sommer/Filip Eftimov

• An improved approach to CIDP
  Claudia Sommer

• Applying the CIDP guidelines: Making an accurate diagnosis
  Filip Eftimov

• Applying the CIDP guidelines: An interactive patient journey
  Claudia Sommer/Filip Eftimov

• Q&A with faculty
  Both speakers

For more information, click here.

Description:
A review of current SMA treatments and how clinical and patient collaboration can provide the missing pieces to optimize outcomes for people with 5q spinal muscular atrophy (SMA)

Learning objectives:
By the end of this session, delegates will:

• Be familiar with an overview of clinical trial data and real-world evidence supporting current 5q SMA therapies
• Appreciate where further research is needed into SMA and its treatments, including concomitant and sequential use of 5q SMA treatments
• Understand how to use individualized outcome measures to identify and incorporate patients’ and carers’ goals into assessments of overall treatment outcomes

Agenda & Speakers:

• 13:00 ‐ 13:05
  Chairperson’s welcome and introduction: What do we still need to know to optimize outcomes in people with 5q SMA?
  Professor Valeria Sansone. Centro Clinico NeMO, Milano, Italy

• 13:05 ‐ 13:25
  Piecing together the evidence: What we have learned and what is missing?
  Professor Julie Parsons. Children’s Hospital Colorado, Aurora, Colorado, USA

• 13:25 ‐ 13:45
  Incorporating Patients’ and carers’ perspectives: Another missing piece of the puzzle?
  Professor Juan F Vázquez Costa. Hospital Universitario de La Fe, Valencia, Spain

• 13:45 ‐ 14:00
  Chairperson-moderated Q&A: What do you think is missing?
  Professor Valeria Sansone. Centro Clinico NeMO, Milano, Italy

For more information, click here.

Biogen have funded and organized this event but have had no other involvement in the ICNMD congress agenda.

The content for this symposium was approved by the Scientific Program Committee as an independent activity held in conjunction with the 17th International Congress on Neuromuscular Diseases. This symposium is not sponsored or endorsed by ICNMD 2022.

Biogen’s treatment for 5q SMA will be discussed, alongside other treatments for 5q SMA. The Summary of Product Characteristics for its product will be available on request from Biogen staff at the event.

Biogen-164071   May 2022

Description:
An opportunity to gain expert insights into the pathophysiology of ALS, the changing treatment landscape, and consider how we might have a positive impact on the patient journey.

Speakers:

• Professor Philip van Damme (Chair)
• Professor Orla Hardiman
• Professor Susanne Petri

For more information, click here.

Objectives:

• Share insights and experiences from NMD and DMD gene therapy clinical trials in relation to site readiness and activation, and how to meet the safety needs for people with NMD and DMD who are receiving gene therapy
• Communicate patient organisation and caregiver insights on the needs of people with DMD in gene therapy clinical trials
• Highlight the role of patient organisations in raising awareness of clinical trials among people with DMD and their caregivers

Agenda:

• 13:00 – 13:05
  Welcome and introduction
  Prof. Dr Nicolas Deconinck

• 13:05 – 13:15
  Introduction to DMD, gene therapies and DMD clinical trials
  Dr Andrés Nascimento Osorio

• 13:15 – 13:30
  Preparing sites for gene therapy clinical trials to ensure clinical and participant needs are met
  Prof. Dr Nicolas Deconinck & Dr Andrés Nascimento Osorio & All faculty

• 13:30 – 13:40
  The role of patient organisations in clinical trials for DMD
   Alejandra Pereda Alonso & All faculty

• 13:40 – 13:50
  Audience Q&A
  All faculty

• 13:50 – 14:00
  Forum summary and close
  Prof. Dr Nicolas Deconinck

Speakers:

• Dr Andrés Nascimento Osorio
  Paediatric Neurologist, Hospital Sant Joan de Déu, Barcelona, Spain

• Dr Nicolas Deconinck
  Principal Investigator, Ghent University Hospital – UZ Gent, Gent, Belgium

• Alejandra Pereda Alonso
  Duchenne Parent Project, Spain

For more information, click here.