Barcelona DMD & SMA Peer-to-Peer Exchange 2026

About the Programme

This comprehensive three-day non-promotional event is dedicated to the management of Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy (SMA). The programme explores the patient journey for both conditions, covering diagnostics, emerging treatment options, site readiness for gene therapy, and future therapeutic expectations.

Designed for high-level neurologists and neuromuscular specialists, this advanced programme chaired by Dr Andrés Nascimento offers a unique opportunity for peer-to-peer exchange. The programme features keynote lectures, interactive case study discussions, and practical insights into Multidisciplinary Team (MDT) disease management.

Delegates will also meet the Sant Joan de Déu (SJD) Neuromuscular Multidisciplinary Team to observe how they practically coordinate and manage complex care pathways for patients with DMD and SMA.

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Expert Faculty

Learn from SJD Barcelona's leading neuromuscular specialists

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Peer-to-Peer Exchange

Interactive case studies & MDT breakout discussions

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Real-World Evidence

Biomarkers, registries, wearables & clinical data

Key Topics

The programme addresses critical areas across both DMD and SMA:

DMD

The Multidisciplinary Team

Diagnostic pathways

Functional scales

Cardiology in DMD

Muscle MRI

Gene therapy & site readiness

Liver & biliary health

The patient perspective

SMA

SMA patient journey

Newborn screening

Real-World Evidence

Treatment options

Bulbar function

Biomarkers, endpoints and wearables

Bone health

Cognitive challenges

Programme Chair

Dr Andrés Nascimento

Dr Andrés Nascimento is the head of the Neuromuscular Diseases (NMD) Unit at Sant Joan de Déu (SJD) Barcelona Children's Hospital, a European reference centre specialising in the diagnosis, treatment and research of Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA).

Operating through a multidisciplinary model of care, the SJD team is actively involved in international clinical trials, natural history studies, and biomarker development. Their approach integrates neurology, rehabilitation, pulmonology, cardiology and psychosocial support to optimise long-term function and quality of life for patients and families.

Programme Agenda

Day 1 – Monday, June 8^th, 2026

Moderator: Dr. Andrés Nascimento

08:30 – 08:45

Welcome & Historical Overview of Sant Joan de Déu Hospital

Andrés Nascimento

08:45 – 09:30

Introducing the Multi-Disciplinary Team Managing DMD & SMA: How the Service is Evolving

Current Diagnostic Pathway for New DMD & SMA Patients and Multidisciplinary Team Monitoring

Carlos Ortez / Andrés Nascimento

09:30 – 10:00

Challenges in Genetic Diagnosis of DMD

Berta Estévez / Daniel Natera

10:00 – 10:30

Factors Influencing Disease Trajectory in DMD

Andrés Nascimento

10:30 – 10:45

Q&A / Discussion

10:45 – 11:00

BREAK

11:00 – 12:00

Practical Implementation of Functional Scales in DMD & SMA

Julita Medina / Obdulia Moya / Sandra Roca

12:00 – 12:30

Liver and Biliary System in DMD & SMA: Diagnosis and Management of Hepatotoxicity

Cristina Molera

12:30 – 13:00

Respiratory and ICU Care in DMD & SMA

Marti Pons

13:00 – 13:15

Q&A / Discussion

13:15 – 14:00

LUNCH

14:00 – 14:30

Bone Health in SMA & DMD: Key Features and Distinctions

Rosa Bou (HSJD)

14:30 – 15:00

Endocrinological Management in DMD

Marta Ramón

15:00 – 15:30

Hub & Spoke Model: Standardizing Procedures, Transition and Continuity of Care in DMD & SMA

Laura Carrera

15:30 – 15:45

Q&A / Discussion

15:45 – 16:00

BREAK

16:00 – 16:45

Breakout Discussion

(1) Multidisciplinary Care Experience, Immune Response Timelines, and What to Measure, When & Why?

(2) Site Readiness for DMD Gene Therapy Dosing — What is Currently Possible in my Hospital and What Are the Key Elements to Prepare?

All Delegates

16:45 – 17:30

Group Feedback & Q&A

Carlos Ortez

Day 2 – Tuesday, June 9^th, 2026

Moderator: Dr. Andrés Nascimento

08:45 – 09:15

Clinical Trials in DMD: Where Do We Stand?

Carlos Ortez

09:15 – 09:45

Gene Therapy in DMD: Vector and Transgene Differences and Their Impact on Genotype, Safety, and Immune Response

Andrés Nascimento / Jésica Expósito

09:45 – 10:30

Preparing Hospitals for Gene Therapy: Requirements and Safety Management in DMD and SMA

Laura Sole / Laura Carrera / Silvia Cerezo

10:30 – 10:45

Q&A / Discussion

10:45 – 11:00

BREAK

11:00 – 11:30

Cardiology in the Care of Patients with DMD: Key Concerns

Sergi Cesar

11:30 – 12:00

Role of Muscle MRI in DMD

Jordi Diaz Manera

12:00 – 12:30

Decoding Duchenne: RNA-Seq Insights into Muscle Variability and Adaptive Response

Jordi Diaz Manera

12:30 – 12:45

Q&A / Discussion

12:45 – 13:30

LUNCH

13:30 – 14:15

The Future of Gene Therapy in Neuromuscular Disorders: What Does the Future Look Like for the Treatment of DMD & SMA?

Eduardo Tizzano

14:15 – 15:15

DMD Case Studies

All Delegates

15:15 – 16:00

Beyond the Muscle: Understanding Neurodevelopment in DMD & SMA from a Neuropsychological Perspective and Its Impact on Emotion and Behaviour

Inés Medina / Irene Zschaeck / Priscila Sánchez

16:00 – 16:30

Patient and Sport

OG (patient) / Irene Zschaeck

16:30 – 17:00

Tour of Facilities

Day 3 – Wednesday, June 10^th, 2026

Moderator: Dr. Andrés Nascimento

08:45 – 09:30

Biomarkers and Genetic Basis of SMA: New Challenges and Insights from SMN2

Andrés Nascimento / Eduardo Tizzano

09:30 – 10:15

Bulbar Function and Nutrition in SMA

Carlos Ruiz / Vanesa Ejarque / Laura Carrera

10:15 – 10:45

Respiratory Function Testing in SMA: Indications for and Management of Non-Invasive Ventilation

María Cols

10:45 – 11:00

Q&A / Discussion

11:00 – 11:20

BREAK

11:20 – 11:50

Emerging Therapeutic Options and Ongoing Clinical Trials in SMA

Jesica Exposito / Laura Carrera

11:50 – 12:20

Integrating Clinical Data and Samples in SMA: The CUIDAME Registry Experience and Biobank Coordination

Stephanie Lotz / Anna Codina

12:20 – 12:50

Beyond the Neuromuscular System: Insights from Pathology Studies

Agustín Luján

12:50 – 13:20

Endpoints, Wearables and Device-Based Monitoring

Daniel Natera / Silvia Cerezo

13:20 – 13:40

Q&A / Discussion

13:40 – 14:30

LUNCH

14:30 – 15:30

SMA Case Studies: Delegates and SJD Team

e.g. 1 × SJD case, 3 delegate cases

All Delegates

15:30 – 16:00

Breakout Discussion: How Would You Increase Diagnosis and Implement Newborn Screening in Your Country?

Andrés Nascimento / Eduardo Tizzano

16:00 – 16:30

Group Feedback & Q&A

16:30 – 16:45

Close of Preceptorship

Andrés Nascimento

Register Now

Spaces are limited for this Peer-to-Peer Exchange.
Please register using the link below:

This meeting is organised by Pi Healthcare & is supported with an unrestricted educational grant from F. Hoffmann-La Roche

Venue: Sant Joan de Déu Hospital | Barcelona, Spain

Barcelona DMD & SMA Peer-to-Peer Exchange 2026

About the Programme

Expert Faculty

Peer-to-Peer Exchange

Real-World Evidence

Key Topics

DMD

SMA

Programme Chair

Dr Andrés Nascimento

Programme Agenda

Day 1 – Monday, June 8th, 2026

Welcome & Historical Overview of Sant Joan de Déu Hospital

Introducing the Multi-Disciplinary Team Managing DMD & SMA: How the Service is Evolving

Challenges in Genetic Diagnosis of DMD

Factors Influencing Disease Trajectory in DMD

Q&A / Discussion

BREAK

Practical Implementation of Functional Scales in DMD & SMA

Liver and Biliary System in DMD & SMA: Diagnosis and Management of Hepatotoxicity

Respiratory and ICU Care in DMD & SMA

Q&A / Discussion

LUNCH

Bone Health in SMA & DMD: Key Features and Distinctions

Endocrinological Management in DMD

Hub & Spoke Model: Standardizing Procedures, Transition and Continuity of Care in DMD & SMA

Q&A / Discussion

BREAK

Breakout Discussion

Group Feedback & Q&A

Day 2 – Tuesday, June 9th, 2026

Clinical Trials in DMD: Where Do We Stand?

Gene Therapy in DMD: Vector and Transgene Differences and Their Impact on Genotype, Safety, and Immune Response

Preparing Hospitals for Gene Therapy: Requirements and Safety Management in DMD and SMA

Q&A / Discussion

BREAK

Cardiology in the Care of Patients with DMD: Key Concerns

Role of Muscle MRI in DMD

Decoding Duchenne: RNA-Seq Insights into Muscle Variability and Adaptive Response

Q&A / Discussion

LUNCH

The Future of Gene Therapy in Neuromuscular Disorders: What Does the Future Look Like for the Treatment of DMD & SMA?

DMD Case Studies

Beyond the Muscle: Understanding Neurodevelopment in DMD & SMA from a Neuropsychological Perspective and Its Impact on Emotion and Behaviour

Patient and Sport

Tour of Facilities

Day 3 – Wednesday, June 10th, 2026

Biomarkers and Genetic Basis of SMA: New Challenges and Insights from SMN2

Bulbar Function and Nutrition in SMA

Respiratory Function Testing in SMA: Indications for and Management of Non-Invasive Ventilation

Q&A / Discussion

BREAK

Emerging Therapeutic Options and Ongoing Clinical Trials in SMA

Integrating Clinical Data and Samples in SMA: The CUIDAME Registry Experience and Biobank Coordination

Beyond the Neuromuscular System: Insights from Pathology Studies

Endpoints, Wearables and Device-Based Monitoring

Q&A / Discussion

LUNCH

SMA Case Studies: Delegates and SJD Team

Breakout Discussion: How Would You Increase Diagnosis and Implement Newborn Screening in Your Country?

Group Feedback & Q&A

Close of Preceptorship

Register Now

Barcelona DMD & SMA
Peer-to-Peer Exchange 2026

Day 1 – Monday, June 8^th, 2026

Day 2 – Tuesday, June 9^th, 2026

Day 3 – Wednesday, June 10^th, 2026