Agenda
Welcome and Introduction
16:45 - 17:00 - Rome time (CET)
Luca Genovese and Maria Rosaria Rollo
President Parent Project aps
I’m here too. Being a sibling of a DMD/BMD patient
17:00 - 18:30 - Rome time (CET)
Speaker: Carla Ferrazzoli, Comitato Siblings Moderators: Marica Pugliese and Anna Petruzza, Parent Project aps
When complexity increases. DMD/BMD and related issues
17:00 - 18:30 - Rome time (CET)
Speakers: Paolo Alfieri and Francesca Cumbo, Ospedale Pediatrico Bambino Gesù, Roma Moderators: Emanuela Giulitti and Sara Billai, Parent Project aps
Motor physiotherapy for kids and adults
17:00 - 18:30 - Rome time (CET)
Speaker: Cristina Bella, Parent Project aps Moderator: Letizia Silicato, Parent Project aps
Session 1: Research projects supported by Parent Project
18:30 - 20:00 - Rome time (CET)
Moderators: Maria Rosaria Rollo and Gloria Antonini - Parent Project aps
Employment of microencapsulated Sertoli Cells as a new tool to treat Duchenne Muscular Dystrophy towards application on patients
Guglielmo Sorci and Sara Chiappalupi – Università di Perugia
Biobanking Urine Cells
Maria Sofia Falzarano – Università di Ferrara
Role of histone deacetylase inhibitor (HDAC) givinostat on cardiac remodeling
Roberto Rizzi – CNR, Institute of Biomedical Technologies (ITB) Milano
Targeting PKC theta to counteract late hallmarks in muscular dystrophy
Marina Bouché – Università di Roma La Sapienza
Epigenetic and transcriptomic profiling of Fibro Adipogenic Progenitors during Duchenne Muscular Dystrophy progression and histone deacetylase inhibitors treatment
Pier Lorenzo Puri and Luca Tucciarone- Sanford Burnham Medical Research Institute, California e Fondazione Santa Lucia, Roma
Questions and answers
The relevance of fundraising activities for Parent Project
20:00 - 20:30 - Rome time (CET)
Maria Rosaria Rollo, Fabiana Ruggiero and Roberto Zoffoli, Parent Project aps
Session 2: Advanced therapies: expectations and reality
16:45 - 18:30 - Rome time (CET)
Moderators: Filippo Buccella and Ilaria Zito - Parent Project aps
Hopes and challenges in advanced therapies
Annemieke Aartsma-Rus, Leiden University Medical Center
Myogenic progenitors from pluripotent stem cells for the treatment of DMD: how far are we
Rita Perlingeiro, University of Minnesota
Development of CRISPR/Cas9-based therapies for Duchenne Muscular Dystrophy
Eleonora Maino, Hospital for Sick Children in Toronto, Canada
How to escape the immune response: current observations and mitigation strategies
Barry Byrne and Manuela Corti, Powell Gene Therapy Center, University of Florida
Questions and answers
Affectivity and sexuality
18:30 - 20:00 - Rome time (CET)
Speaker: Max Ulivieri - Comitato Love Giver
Moderators: Marica Pugliese and Giada Perinel, Parent Project aps
What we eat matters. Nutritional aspects in DMD and BMD
18:30 - 20:00 - Rome time (CET)
Speaker: Simona Bertoli - Università degli studi di Milano
Moderator: Maria Capece, Parent Project aps
Laws, rights and bureaucracy - Italian only
18:30 - 20:00 - Rome time (CET)
Speaker: Carlo Giacobini - journalist Moderators: Nicole Coluzzi and Carmen Sileo, Parent Project aps
Presentation of the new video animation “What are clinical trials?”
16:30 - 17:00 - Rome time (CET)
Luca Genovese and Marica Pugliese, Parent Project aps
Session 3: Addressing dystrophin production: from DMD/BMD diagnosis to gene therapy
17:00 - 18:30 Rome time (CET)
Moderators: Fernanda De Angelis and Ilaria Zito, Parent Project aps
DMD/BMD diagnosis
Eugenio Mercuri, Policlinico Gemelli – Roma
CIFFREO: Pfizer’s Phase 3 study of gene therapy for DMD
Beth Belluscio, Pfizer
IGNITE DMD Clinical Trial Update
Carl Morris, Solid Biosciences
Micro-dystrophin gene therapy
Alex Murphy, Roche – Teji Singh, Sarepta
An Introduction to REGENXBIO and RGX-202, a new gene therapy program for Duchenne Muscular Dystrophy (DMD)
Olivier Danos, REGENXBIO
Questions and answers
Session 4: Psychological aspects in the communication of diagnosis: the family perspective
18:30 - 20:00 - Rome time (CET)
Moderators: Emanuela Giulitti and Nicole Coluzzi, Parent Project aps
Session 5: Genetic matters
09:45 - 12:00 - Rome time (CET)
Moderators: Filippo Buccella, Gloria Antonini and Ilaria Zito, Parent Project aps
As genetic diagnosis addresses personalized therapies
Alessandra Ferlini, Università di Ferrara
RNA-Targeted Therapies: PMO & PPMO update
Luigi Picaro, Sarepta Therapeutics
NS Pharma Inc and the Viltolarsen Clinical Development Program
Benjamin Yungher, NS Pharma
Dyne Therapeutics: Committed to Delivering Transformative Therapies for Duchenne Muscular Dystrophy Patients
Romesh Subramanian, Dyne Therapeutics
Mesoangioblast mediated exon skipping
Giulio Cossu, Università di Manchester
Nonsense mutations
Luca Bello, Università degli Studi di Padova
Questions and answers
Session 6: Clinical and Drug managment: present and future
15:45 - 18:00 - Rome time (CET)
Moderators: Fernanda De Angelis and Gloria Antonini, Parent Project aps
Clinical management
Adele D’Amico, Ospedale Pediatrico Bambino Gesù – Roma
Phase 3 PolarisDMD trial of edasalonexent in Duchenne
Joanne M. Donovan, Catabasis Pharmaceuticals
Vamorolone as a potential safer alternative to corticosteroids in DMD
Eric Hoffman, ReveraGen BioPharma
Pamrevlumab Program Development in DMD
Bassem Elmankabadi, FibroGen
An update of Givinostat clinical development
Paolo Bettica, Italfarmaco
Establish clinical and genetic characteristics in young, steroid naïve subjects with Duchenne muscular dystrophy: the FOR DMD study
Michela Guglieri, Newcastle University
Digital endpoints in rare disease: measuring ease of movement in Duchenne Muscular Dystrophy
Mindy Leffler, Casimir
Questions and answers
Session 7: Psychological aspects of the transition from childhood to adolescence: the family perspective
18:00 - 19:15 - Rome time (CET)
Moderators: Tiziana D'Auria and Marica Pugliese, Parent Project aps
By integrating we learn: school integration - Italian Only
10:00 - 11:30 - Rome time (CET)
Ppeaker: Giancarlo Onger – C.N.I.S. Brescia
Moderators: Tiziana D'Auria and Lucrezia Salucci, Parent Project aps
Easy life. Home automation at the service of the community
10:00 - 11:30 - Rome time (CET)
Speaker: Luca Bertazzoni – Domotica Quadrifoglio
Moderators: Stefania Manganaro and Letizia Sticca, Parent Project aps
Session 8: Family guidelines and TRIALS for BMD
11:30 - 13:00 - Rome time (CET)
Moderators: Gerardo Laurenzana, Antonio Bellon and Fernanda De Angelis, Parent Project aps
Developing family guidelines for BMD
Elena Pegoraro, Università degli Studi di Padova
Givinostat in BMD
Giacomo Comi, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico, Milano
A Mitochondrial Approach to Becker Muscular Dystrophy
Ransi Somaratne, Epirium Bio
Introduction to Edgewise Therapeutics: Our company and our plans
Alan Russel, Edgewise Therapeutics
Questions and answers
Session 9: Psychological aspects of adult life: the patient perspective
15:00 - 16:30 - Rome time (CET)
Moderators: Nicole Coluzzi and Maria Capece, Parent Project aps
Session 10: Heart and lung function
16:30 - 19:00 - Rome time (CET)
Moderators: Filippo Buccella and Gloria Antonini, Parent Project aps
Final farewell
19:00 - 19:30 - Rome time (CET)
Luca Genovese, Parent Project aps
Cardiac managment
Rachele Adorisio, Ospedale Pediatrico Bambino Gesù – Roma
Respiratory management
Fabrizio Racca, Azienda Ospedaliera SS Antonio Biagio e Cesare Arrigo di Alessandria
Santhera: Learnings from the SIDEROS trial and enduring commitment to the Duchenne community
Jodi Wolff, Santhera Pharmaceuticals
The FIGHT DMD Trial
Ines Macias-Perez, Cumberland Pharmaceuticals
CAP-1002 (cardiac cell therapy) for the treatment of upper limb and cardiac function in later stage DMD patients
Linda Marbán, Capricor Therapeutics
Emergency Card
Fabrizio Racca, Azienda Ospedaliera SS Antonio Biagio e Cesare Arrigo di Alessandria
Questions and answers