The 19th edition of the International Conference on Duchenne and Becker Muscular Dystrophy (DMD / BMD) will be streamed from Thursday 17 to Sunday 20 February 2022

A new edition of this event, full of opportunities for information and study for the entire Duchenne and Becker community, is coming. For the second consecutive year the event will be delivered online; recent developments in the covid-related  situation have led to the choice of a solution that fully protects the safety of all people in the patient community.

The claim of the 2022 conference will be Journey to the future: drawing new maps, to represent the uninterrupted orientation of the Duchenne and Becker community towards progress in science and in the social sphere. In recent decades, this great network  has begun to open roads that did not yet exist and, over time, the maps that guide us are always evolving. Research takes steps forward, expanding the map of shared knowledge. At the same time, the scenario of Duchenne and Becker gradually continues to change and horizons widen: today’s children and young people are called to make plans and to design their own personal path in the world.

Agenda

16:45 - 17:00 ROME TIME (CET)

Welcome and introduction

17:00 - 18:30 Sessioni Parallele

I’m here too. Being a sibling of a DMD/BMD patient

Laura Gentile, Psicologa e Psicoterapeuta. Responsabile Scientifico Progetto Rare Sibling – Omar

A journey through time: BMD, between experiences and life projects

Giada Perinel - Emanuela Giulitti - Antonio Bellon, Parent Project aps

Integrating and learning: school integration (Italian only)

Giancarlo Onger, C.N.I.S. Brescia

Laws, rights and bureaucracy (Italian only)

Carlo Giacobini, Giornalista e divulgatore

16:00 - 17:30 ROME TIME (CET)

LOOKING AHEAD IN THE LANDSCAPE OF RESEARCH FOR DUCHENNE/BECKER

Moderazione: I. Zito, L. Genovese

Genome Editing for Duchenne muscular dystrophy

Charles Gersbach

New human muscle disease models to study muscular dystrophies and test therapies

Francesco Saverio Tedesco, UCL, GOSH & The Francis Crick Institute, London

HDACi treatment to correct communication between FAPs and Muscle Stem cells and counteract DMD pathology 

Martina Sandonà, IRCCS Fondazione Santa Lucia, Roma 

Microencapsulated Sertoli cells in Duchenne muscular dystrophy. Potentials for application on patients

Guglielmo Sorci and Sara Chiappalupi, Università di Perugia

17:30 - 19:30 ROME TIME (CET) Sessioni Parallele

Easy life. La domotica al servizio della Comunità e presentazione del progetto Camera Libera tutti

Luca Bertazzoni, Quadrifoglio Impianti - Daniele Khalousi, Relazioni Esterne FabLab Parma

Respiratory physiotherapy for kids and adults (Italian only)

Cristina Bella, Parent Project aps

When complexity increases. DMD/BMD and related issues

Paolo Alfieri - Francesca Cumbo, Ospedale Pediatrico Bambino Gesù, Roma

What we eat matters. Nutritional aspects in DMD and BMD

Simona Bertoli, Università degli Studi di Milano

10:00 - 12:00 ROME TIME (CET)

RESEARCH PROJECTS SUPPORTED BY PARENT PROJECT

Moderazione: G. Antonini, P. De Leo

Counteracting inflammation in DMD by promoting pro-regenerative macrophage polarization through a metabolic approach

Elisabetta Ferraro, Università degli Studi di Pisa

A novel epigenetic approach to ameliorate muscle homeostasis in Duchenne Muscular Dystrophy

Giuseppina Caretti, Università degli Studi di Milano

Pilot study on drug-mediated Nfix inhibition as a new therapy for Muscular Dystrophies

Graziella Messina, Università degli Studi di Milano 

Phenotypic variability in Becker Muscular Dystrophy: proposal of a clinical and molecular characterization protocol to stratify patients towards trial readiness

Gabriele Siciliano, AOU Pisana – Ospedale Santa Chiara, Pisa 

Cardiomyopathy due to dystrophinopathies: how it is characterized and it progresses

Rachele Adorisio, Ospedale Pediatrico Bambino Gesù, Roma

12:00 - 13:00 ROME TIME (CET)

Poster Session

Moderazione: G. Antonini, F. De Angelis, I. Zito

14:00 - 16:30 ROME TIME (CET)

RESTORING DYSTROPHIN PRODUCTION, PART I: GENE THERAPY

Moderazione: F. De Angelis, F. Buccella

Overview on clinical results of gene therapy trials 

Eugenio Mercuri, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Roma

Updates on Fordadistrogen Movaparvovec trials for Duchenne

Beth Belluscio, Pfizer 

Delandistrogene moxeparvovec (SRP-9001) micro-dystrophin gene therapy, Roche and Sarepta update 

Valeria Lovato, Roche 

IGNITE DMD SGT-001 Microdystrophin Gene Therapy Clinical Trial update 

Roxana Donisa Dreghici, Solid Biosciences 

REGENXBIO:  An update on RGX-202, an Investigational Gene Therapy for Duchenne Muscular Dystrophy (DMD) 

Olivier Danos, RegenXbio 

Genethon AAV8 microdystrophin gene therapy for DMD

Francesco Muntoni, Dubowitz Neuromuscular Centre, London

Gene therapy for DMD exon 2 duplication

Kevin Flanigan, Nationwide Children’s Hospital, Ohio 

Ex vivo gene Therapy 

Giulio Cossu, University of Manchester

16:00 - 17:30 ROME TIME (CET) Round table

Building an INDEPENDENT LIFE: the sooner the better!

Moderation: A. Petruzza e G. Perinel, Parent Project aps

17:30 - 19:30 Rome Time (CET)

Update on RNA-Targeted Therapies: PMO and PPMO 

Lorna Hill and Jon Tinsley, Sarepta Therapeutics 

Updates on Viltepso (viltolarsen)

Robert Crozier, NS-Pharma 

Transforming the Treatment Paradigm for Duchenne Muscular Dystrophy

Ash Dugar, Dyne Therapeutics

Advancing Exon Skipping AOCs for the Treatment of Duchenne Muscular Dystrophy 

Mark Stahl, Avidity Biosciences

Unlocking the potential of oligonucleotide therapeutics 

James McArthur, PepGen

Nonsense mutations

E. Mercuri, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome

RESTORING DYSTROPHIN PRODUCTION, PART II: GENE CORRECTION

Moderazione: I. Zito, E. Magnano

18:00 - 19:30 ROME TIME (CET) Parallel Session

Affectivity and sexuality: a moment of confrontation

Maximiliano Ulivieri - Comitato Love Giver

10:30 - 12:30 ROME TIME (CET) Round Table

The impact of Covid on daily life, constraints and perspectives

Data on a COVID survey will be presented and through the testimonies of boys, families and operators we will discuss the most relevant topics emerged. Aspects of daily life will also be discussed, from clinical management, through physiotherapy, to school and social participation.
Highlights on services of telecardiology with Dr. Villano and tele-physiotherapy with Dr. Bella will follow.

14:00 - 15:00 - Rome Time (CET)

THE IMPORTANCE OF PJ NICHOLOFF PROTOCOL AND THE EMERGENCY CARD PROJECT

F. Buccella, P. Furlong, Fabrizio Racca

15:00 - 16:30 Rome Time (CET)

CLINICAL TRIALS IN BECKER MUSCULAR DYSTROPHY

Moderation: F. De Angelis, G. Laurenzana

Givinostat in Becker 

Sara Cazzaniga, Italfarmaco

Phase II pilot study with vamorolone vs placebo in Becker muscular dystrophy

Elena Pegoraro, Università di Padova

Updates on EDG-5506

Joanne Donovan, Edgewise

16:30 - 18:30 Rome Time (CET)

IMPROVING MUSCLE HEALTH, OPPOSING INFLAMMATION AND FIBROSIS

Moderation: G. Antonini, S. Capponi

Updates on vamorolone in DMD 

Eric Hoffman, ReveraGen BioPharma

ATL1102, Targeting Inflammation in DMD and Clinical Development Update

Annabell Leske, Antisense Therapeutics 

Givinostat. clinical trials update in DMD

Paolo Bettica, Italfarmaco 

Updates on pamrevlumab trials for Duchenne

Ewa Carrier, FibroGen 

The FIGHT DMD Trial – An FDA Sponsored Study Aimed to Impact the Cardiomyopathy associated with DMD 

Ines Macias-Perez, Cumberland Pharmaceuticals 

CAP-1002, a Therapeutic for the Treatment of Non-Ambulant DMD

Linda Marban, Capricor Therapeutics