World Conference on PCDH19

Scientific research status and therapeutic perspectives

Third Edition - 23-24 October 2015 - via Matera, 18 - Rome

Pre-conference Meeting*, Friday 23rd, 4 pm - 7 pm

Preliminary data and potential challenges in the first PCDH19 clinical trial
Chair Joseph Sullivan

Neurosteroids and PCDH19 function: Allopregnanolone and beyond
Chair Jozef Gecz

Understanding the PCDH19 role in neuronal development: What help from pluripotent stem cells (IPSCs) and animal
Chair Jack Parent

New horizons: Cannabidiol as a potential treatment for (PCDH19) uncontrolled epilepsy and associated behavioral
Chair Nicola Specchio

(*) for researchers and clinicians, by invitation only

Conference, Saturday 24th,  9.30 am - 6 pm

9.30 Registration of Participants

9.50 Welcome Speech - Francesca Squillante (Onlus Insieme per la Ricerca PCDH19)

Genetics - Moderators Patrizia D’Adamo (IRCCS Ospedale San Raffaele) and Renzo Guerrini
(Ospedale Pediatrico Meyer di Firenze)

10.00 Maria Passafaro (Istituto di Neuroscienze, CNR Milano) - Molecular Characterization of PCDH19 in
Rat Hippocampal Neurons

10.20 Jack Parent (University of Michigan) - Modeling PCHD19 Epilepsy with Patient-Derived Cells and Rat In
Utero Electroporation

10.40 Claudia Compagnucci (Ospedale Pediatrico Bambino Gesù di Roma) - PCDH19 and the
Establishment of Planar Cell Polarity in IPS-derived Developing Neurons in an X-linked Model of Epilepsy

11.00 Alessandra Terracciano (Ospedale Pediatrico Bambino Gesù di Roma) - Mosaic PCDH19 Point
Mutation in Male Patients

11.20 Jozef Gecz (University of Adelaide) - PCDH19; From Mutation to Molecular Mechanism(s) to Treatment.
Are We There Yet?

11.40 Coffee Break

Therapeutic perspectives - Moderators Federico Vigevano (Ospedale Pediatrico Bambino Gesù
di Roma) and Prof. Bernardo Dalla Bernardina (Azienda Ospedaliera Universitaria di Verona)

12.00 Giuseppe Biagini (Università degli Studi di Modena e Reggio Emilia) - Neurosteroids as Endogenous
Modulators of Epileptic Seizures

12.20 Patroneva Albena (Marinus Pharmaceuticals, Inc.) - Introduction of ganaxolone as a treatment for
PCDH19 female pediatric epilepsy

12.40 Joseph Sullivan (UCSF Pediatric Epilepsy Center)- Potential New Treatment Options for PCDH19

13.00 Stephen Wright (GW Pharmaceuticals) - Cannabidiol in Treatment resistant epilepsy in children. Can we
extrapolate the data to children with PCDH19?

13.20 Pasquale Striano (Istituto G. Gaslini, Università di Genova) - Alternative Treatments for Drug-Resistant

13.40 Lunch

14.45 Round Table - Moderator Nicola Specchio (Ospedale Pediatrico Bambino Gesù di Roma) :
Norimichi Higurashi ( School of Medicine of Tokyo ) ; Eric Leguern (Hospital Pitié-Salpétrière, Paris);
Raffaella Cusmai (Ospedale Pediatrico Bambino Gesù di Roma)

16.30- 18.00 Questions & Answers

Register to attend the Conference

With the support of:

with support of Telethon, NRCI, Bambino Gesù Ospedale Pediatrico, SINPIA, eCampus