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XII INTERNATIONAL CONFERENCE ON DUCHENNE AND BECKER MUSCULAR DYSTROPHY 21 – 23 February 2014 Ergife Palace Hotel, Rome - Italy Temporary Program Friday, February 21 14:00

Registration

14:30

Welcome and introduction Filippo Buccella- Parent Project onlus, Roma, Italia

14:40 – 16:10 DBMD Clinical Management 0-14 years Chair: Nathalie Goemans- Dept. of Pediatrics and Child Neurology, University Hospitals, Leuven, Belgium

DBMD Guide lines Nathalie Goemans- Dept. of Pediatrics and Child Neurology, University Hospitals, Leuven, Belgium

Clinical Diagnosis Tiziana Mongini - Dip. Di Neuroscienze Università di Torino, Ospedale Molinette, AUS. Giovanni Battista, Torino, Italia

DMD Molecular Diagnosis: past, present and future Filippo Santorelli - Medicina Molecolare e Malattie Neuromuscolari, IRCCS Stella Maris, Pisa, Italia

Steroids and health examinations Adele D’Amico - Unità di Malattie Neuromuscolari e Neurodegenerative, Dip. di neuroscienze, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italia

• •

Physiotherapy Elena Mazzone - Dip. Neuropsichiatria Infantile, Policlinico Agostino Gemelli, Roma, Italia Cardiac management Rachele Adorisio - Dip. Medico Chirurgico di Cardiologia Pediatrica, U.O. Cardiologia, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italia

Pulmonary Management Fabrizio Racca - Rep. di Anestesia Pediatrica AO Nazionale, SS. Antonio e Biagio e Cesare Arrigo di Alessandria, Alessandria, Italia

Emotional and cognitive aspects in DMD. The importance of communication of the diagnosis. Emanuela Giulitti - Parent Project onlus, Roma, Italia

16:10 Question & Answer SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


16:40 – 17:40 anti-inflammatory strategies Chair: Kanneboyina Nagaraju- Research Center for Genetic Medicine, Children's National Medical Center (CGMR), Washington, USA

Overview on anti-inflammatory strategies Kanneboyina Nagaraju- Research Center for Genetic Medicine, Children's National Medical Center (CGMR), Washington, USA

VBP-15 Kanneboyina Nagaraju- Research Center for Genetic Medicine, Children's National Medical Center (CGMR), Washington, USA

Flavocoxid: pilot clinical study in DMD Sonia Messina - Dip. Di Neurologia e Malattie Neuromuscolari, Policlinico Universitario Messina, Messina, Italia

FOR DMD: Double-blind randomized trial to optimize steroid regime in DMD Michela Guglieri - Institute of Genetic Medicine, Newcastle University, Newcastle upon Tyne, Great Britain

Risk factors for bone loss and fractures in boys affected by DMD: from genetic to treatment (RisBo) Maria Luisa Bianchi - Centro Malattie Metaboliche Ossee, Istituto Auxologico Italiano IRCCS, Milano, Italia

17:40 Question & Answer

Saturday, February 22 8:00

Registration

8:40

Welcome and introduction Filippo Buccella- Parent Project onlus, Roma, Italia

8:50 – 9:50 Cell Therapy Chair: Giulio Cossu - Institute of Inflammation and Repair, University of Manchester, Manchester, Great Britain

Overview on cell therapy Giulio Cossu - Institute of Inflammation and Repair, University of Manchester, Manchester, Great Britain

Biomaterials for Delivery Pier Lorenzo Puri - Sanford/Burnham Medical Research Institute, La Jolla USA & Fondazione Santa Lucia, Roma, Italia

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


Human Artificial Chromosomes and iPS cells for DMD therapies Francesco S. Tedesco - Dept. of Cell and Developmental Biology, University College, London, Great Britain

From skin to skeletal muscle: a potential for autologous transplantation in DMD Rita Perlingeiro - Dept. of Medicine, Lillehei Heart Institute, University of Minnesota, Minneapolis, USA

Allotransplantation of Mesoangioblast for Duchenne Muscular Dystrophy: a phase 1/2a clinical trial Giulio Cossu - Institute of Inflammation and Repair, University of Manchester, Manchester, Great Britain

9:50 Question & Answer 10:20– 11:10 Correction Strategies Chair: Annemieke Aartsma-Rus - Dept. of Human Genetics, Leiden University Medical Center LUMC, Leiden, The Netherlands

Overview on correction strategies Annemieke Aartsma-Rus - Dept. of Human Genetics, Leiden University Medical Center LUMC, Leiden, The Netherlands

Biglycan as a candidate therapeutic for all forms of DMD Justin Fallon - Neuroscience Department of Brown University, Providence, USA • Genome Editing for the Correction of Duchenne Muscular Dystrophy Charles Gersbach - Dept. of Biomedical Engineering, Duke University, Durham, USA • Update on the Clinical Development of Ataluren in DMD Allen Reha - PTC Therapeutics •

11:10 Question & Answer

11:40 – 13:05 Exon Skipping Strategies Chair: Francesco Muntoni - Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Deseases UCL Institute of Child Health and Great Ormond Street Hospital for Children (GOSH), Dept. Of Developmental Neurocience, London, Great Britain

Overview on exon skipping strategies Francesco Muntoni - Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Deseases UCL Institute of Child Health and Great Ormond Street Hospital for Children (GOSH), Dept. Of Developmental Neurocience, London, Great Britain

Optimizing exon skipping in animal models

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


Annemieke Aartsma-Rus - Dept. of Human Genetics, Leiden University Medical Center LUMC, Leiden, The Netherlands

Exon skipping for duplications Kevin Flanigan - Center for Gene Therapy, Nationwide Children’s Hospital, Ohio, USA • Multi-exon skipping for DMD Toshifumi Yokota - Dept. of Medical Genetics, University of Alberta Faculty of Medicine and •

Dentistry, Edmonton, Alberta, Canada

Prosensa exon skipping trials update Giles Campion – Prosensa • Drisapersen exon skipping trials update Giles Campion – Prosensa • Update on the Eteplirsen Muscular Dystrophy Program Petra Duda - Sarepta Therapeutics •

13:05

Question & Answer

13:35

Lunch

14:40 – 16:10 DBMD Clinical Management over 14 years Chair: Douglas Biggar- University of Toronto, Holland Bloorview Kids, Rehabilitation Hospital, Toronto, Canada

Clinical Menagement of adults with Duchenne Muscular Dystrophy Douglas Biggar- University of Toronto, Holland Bloorview Kids, Rehabilitation Hospital, Toronto, Canada

DBMD diagnosis and management over 14 yrs: guidelines Irene Bruno - U.C.O. Clinica Pediatrica, IRCCS materno infantile Burlo Garofalo di Trieste, Italia

Physiotherapy Elena Mazzone - Dip. Neuropsichiatria Infantile, Policlinico Agostino Gemelli, Roma, Italia • Cardiac management Piergiorgio Bruno - Dip. Scienze Cardiovascolari, Policlinico Agostino Gemelli, Roma, Italia • Managing Cardiac emergencies Antonio Amodeo - Ospedale Pediatrico Bambino Gesù, Roma, Italia • Pulmonary Management Grazia Crescimanno - Istituto di Biomedicina e Immunologia Molecolare CNR, Palermo, •

Italia

Managing Respiratory emergencies Fabrizio Racca - Rep. Anestesia Pediatrica AO NAzionale SS Antonio e Biagio Cesare Arrigo di Alessandria, Alessandria, Italia

Becoming adults: autonomy and quality of life Maria Caterina Pugliese - Parent Project onlus, Roma, Italia

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


16:10 Question & Answer

20:00 Dinner

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


Sunday, February 23 9:00 – 9:30 “Audit Civico” in Duchenne and Becker Muscular dystrophy, state of the art Filippo Buccella- Parent Project onlus, Roma, Italia 9:30

Patient Registry and research funding: where we are going Fernanda De Angelis – Parent Project onlus, Roma, Italia

9:40

Search for Genetic modifiers of Duchenne Muscular Dystrophy Stan Nelson – David Geffen School of Medicine at UCLA, Los Angeles, USA

10:00 – 10:50 Outcome measure research Chair: Eugenio Mercuri - Dip. Neuropsichiatria Infantile, Policlinico Agostino Gemelli, Roma, Italia

Overview on outcome measure research Eugenio Mercuri - Dip. Neuropsichiatria Infantile, Policlinico Agostino Gemelli, Roma, Italia • Outcome measure Craig McDonald - Dept. of Physical Medicine and Rehabilitation, UC Davis Children’s •

Hospital, CA, USA

Molecular Biomarkers Pietro Spitali - Dept. of Human Genetics, Leiden University Medical Center LUMC, Leiden, The Netherlands

Upper Limb Eugenio Mercuri - Dip. Neuropsichiatria Infantile, Policlinico Agostino Gemelli, Roma, Italia

10:50 Question & Answer

11:20 – 12:20 Improving muscle mass and health Chair: Michael Rudnicki- Ottawa Hospital Research Institute, Dept. of Medicine and Department of Cellular and Molecular Medicine, Faculty of Medicine, University of Ottawa, Ottawa, USA

Overview on Improving muscle mass and health Michael Rudnicki- Ottawa Hospital Research Institute, Dept. of Medicine and Department of Cellular and Molecular Medicine, Faculty of Medicine, University of Ottawa, Ottawa, USA

Wnt7a as a potential therapy for muscular dystrophy Michael Rudnicki- Ottawa Hospital Research Institute, Dept. of Medicine and Department of Cellular and Molecular Medicine, Faculty of Medicine, University of Ottawa, Ottawa, USA

Givinostat Trial Paolo Bettica - Italfarmaco SpA • ISOFEN •

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


Emilio Clementi- Unità di Farmacologia Clinica, Ospedale Luigi Sacco, Università di Milano, Milano, Italia

Tadalafil trial in DMD Michele Guadagna - Neuroscience- Eli Lilly Italia

12:20 Question & Answer

SEDE LEGALE E OPERATIVA: Via N. Coviello, 12/14 00165 Roma

tel. 06 66182811 – fax 06 66188428 Centro Ascolto Duchenne 800 943 333 partita iva 05203531008 - www.parentproject.it


pp-conf-2014-draft-agenda-upd-03-02