XVIII INTERNATIONAL CONFERENCE ON DUCHENNE AND BECKER MUSCULAR DYSTROPHY

February 17-21, 2021

Alone we imagine the future, together we grow

Many paths of research towards one direction
The 18th edition of Parent Project’s International Conference on Duchenne and Becker Muscular Dystrophy will take place online from the 17th to the 21st of February, 2021.

Last year’s edition had to be cancelled due to the Covid-19 outbreak; this year the event will be back, although based on a web platform, to provide up-to-date scientific information and much more for the entire Duchenne and Becker community.

The 2021 conference claim will be “Alone we imagine the future, together we grow” with the subtitle “Many paths of research towards one direction”, to underline the fundamental dynamics of convergence and interrelation between the many actors who make up the Duchenne community: patients, family members, researchers, clinicians, companies, volunteers.

Agenda

Welcome and Introduction

16:45 - 17:00 - Rome time (CET)

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

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

XVIII INTERNATIONAL CONFERENCE ON DUCHENNE AND BECKER MUSCULAR DYSTROPHY

17 21 febbraio 2021