Due to a temporary technical problem that does not depend on us, the Conference website is not 100% functional at the moment.
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.
You can also follow the live stream on Facebook (in Italian) and on our YouTube channel (in English).
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