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You can still join us, just going on the website (https://conferenza.parentproject.it/) and clicking on the LIVE STREAM button. You will redirected to Zoom.

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Here you can find today and tomorrow’s agenda.

XIX INTERNATIONAL CONFERENCE ON DUCHENNE AND BECKER MUSCULAR DYSTROPHY

February 19 

SATURDAY, FEBRUARY 19TH   

10:00 SESSION 2 RESEARCH PROJECTS SUPPORTED BY PARENT PROJECT

Introduction: G. Antonini 

Moderation: 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 

11:15 Q&A

12:00 POSTER SESSION

13:00 Lunch break

14.00 SESSION 3 RESTORING DYSTROPHIN PRODUCTION, PART I: GENE THERAPY

Introduction: F. De Angelis

Moderation: 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

15:30 Q&A

16:00 SESSION 4 ROUND TABLE Building an INDEPENDENT LIFE: the sooner the better! 

The process of building an independent life develops during the different phases of the life cycle: childhood, adolescence and adulthood. The theme will be discussed with testimonials from parents, boys and experts involved in this path. 

Introduction and moderation: A. Petruzza and G. Perinel, Parent Project aps

17:30 SESSION 5 RESTORING DYSTROPHIN PRODUCTION, PART II: GENE CORRECTION

Introduction: I. Zito

Moderation: I. Zito, E. Magnano

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

18:45 Q&A

18:00 PARALLEL SESSION 

Affectivity and sexuality: a moment of confrontation

Massimiliano Ulivieri, Comitato Love Giver

19:30 CLOSE

SUNDAY, FEBRUARY 20TH 

10:30 SESSION 6 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.

Introduction and moderation: Emanuela Giulitti, Maria Caterina Pugliese e Tiziana D’Auria, Parent Project aps

12:30 Lunch break

14:00 THE IMPORTANCE OF PJ NICHOLOFF PROTOCOL AND THE EMERGENCY CARD PROJECT

Introduction: F. Buccella, P. Furlong

Emergency Card: work in progress – The importance of PJ Nicholoff protocol

Fabrizio Racca, Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo, Alessandria

15:00 SESSION 7 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 

15:30 Q&A

16:30 SESSION 8 IMPROVING MUSCLE HEALTH, OPPOSING INFLAMMATION AND FIBROSIS

Introduction: G. Antonini

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 

18:00 Q&A

18:30 CLOSE