Preliminary Family Program

Friday, July 31, 2020

07:30-09:00 Sponsored Breakfast Symposium by ORCHARD THERAPEUTICS
09:00-09:30 WELCOME SPEECH
MPS Spain & representative from the Ministry of Health or Local Government
09:30-10:15 OPENING KEYNOTE SPEECH
GENERAL OVERVIEW ON NEWBORN SCREENING FOR MPS
Prof. Dr. Frits Wijburg (Netherlands)
10:15-10:45 COFFEE BREAK
10:45-11:15 CLINICAL TRIALS OVERVIEW & EVOLUTION
Dr. Emil Kakkis (USA)
CLINICAL TRIALS & PATIENT'S INVOLVEMENT PART 1
Moderators: Dr. Maria Escolar (USA) & Mr. John Crowley (USA)
11:15-11:25 European Reference Network
Dr. Maurizio Scarpa, Coordinating Center for Rare Diseases (Italy)
11:25-12:35 Patient Advocate
Ms. Michaela Weigl, MPS Austria (Austria)
12:35-11:45 Adult Patient
Ms. Barbara Mamatis, Greek Solidarity Association (Greece)
11:45-11:55 European Federation of Pharmaceutical Industries and Associations
To be determined
11:55-12:30 Open Discussion Session
12:30-14:00 LUNCH
CLINICAL TRIALS & PATIENT'S INVOLVEMENT PART 2
Moderators: Dr. Francesc Palau (Spain) or Dr. Josep Torrent (Spain)
14:00-14:10 Drug development
To be determined
14:10-14:20 End points & patient involvement
To be determined, MPS International
14:20-14:30 Registries
Dr. Josep Torrent-Farnell, Drug Department Catalan Government (Spain)
14:30-14:40 Challenges of reimbursement
To be determined, Eurordis
14:40-14:50 Administration
Dr. César Hernández García, AEMPS & EMA (Spain)
14:50-15:30 Open Discussion Session
15:30-16:00 COFFEE BREAK
HEART, BONE & GROWTH HORMONE, NEUROSURGERY, PNEUMOLOGY
Moderator: Dr. Montserrat Morales (Spain)
16:00-16:25 Topic: Heart
MD. Prof. Christoph Kampmann (Germany)
16:25-16:50 Topic: Bone & Growth Hormone
MD PhD Shunji Tomatsu (USA)
16:50-17:15 Topic: Neurosurgical Management of MPS
FRCS (SN) Dr. Guirish A. Solanki (UK)
17:15-17:40 Topic: Pneumology
MD. Dr. David Rapoport (USA)
17:40-18:00 Q&A Session
18:00-19:00 EXHIBITION AREA & POSTER SESSION
19:00-21:00
REMEMBRANCE CEREMONY

Saturday, August 1, 2020

BEST PRACTICES INCLINICAL MANAGEMENT
Moderators: Dr. Kim Hemsley (Australia) & Dr. Gregory Pastores (Ireland)
08:30-09:00 Topic: Clinical management & optimal care
Dr. Nicole Muschol (Germany)
09:00-09:30 Topic:Multidisciplinary team approach
Dra. Pineda’s Team at Sant Joan de Déu Hospital, Barcelona (Spain)
09:30-10:00 Topic: Inter center coordination
Dr. Eloisa Teles (Portugal)
THE GLOBAL VOICE OF PATIENTS
10:00-10:10 Marija Joldic, MPS Europe
10:10-10:20 Virginia Tsai, MPS Taiwan
10:20-10:30 Regina Prospero, MPS Brazil
10:30-11:00 COFFEE BREAK
IMPORTANT ISSUES IN TREATMENT
Moderators: to be determined
11:00-11:20 Topic: Non-invasive ventilation
Dr. Ana Voth (Spain)
11:20-11:40 Topic: Anesthesia/ airways
Dr. Enric Monclús (Spain)
11:40-12:00 Topic: Orthopedics/technical help
Dr. Bianca Link (Switzerland)
12:00-12:20 Topic: Rehabilitation / physiotherapy
To be determined
12:20-12:40 Topic: Home Therapies/on-the-go treatment
To be determined
12:40-14:00 LUNCH
GENE THERAPIES
Moderators: Dr. Benedicte Heron (France) & Dr. Mª Luz Couce (Spain)
14:00-14:25 To be determined
14:25-14:50 To be determined
14:50-15:15 To be determined
15:15-15:40 To be determined
15:40-16:00 Q&A Session
16:00-16:20 COFFEE BREAK
NEW THERAPIES
Moderators: Dr. Joseph Muenzer (Germany) & Dr. Mireia del Toro (Spain)
16:20-16:40 Topic: Odiparcil
Dr. Nathalie Guffon (France)
16:40-17:00 Topic: Trehalosa
Dr. Marco Ardielo
17:00-17:20 Topic: PPS
Dra. Calongera Simonaro (USA)
17:20-17:45 Q&A Session
17:45-19:00 EXHIBITION AREA & POSTER SESSION
21:00-23:00 CONGRESS DINNER

Sunday, August 2, 2020

RELATED DISEASES
Moderators: to be determined
08:30-08:55 Topic: Mucolipidosis
Dr. Thomas Braulke (Germany)
08:55-09:20 Topic: New MPS VPS33A
Dr. Timothy Cox (UK)
09:20-09:45 Topic: Sialidosis
Dr. Alessandra D’Azzo (USA)
09:45-10:15 Q&A Session
10:15-10:45 COFFEE BREAK
QUALITY OF LIFE - WHAT WE NEED
Moderators: To be determined
10:45-10:55 Psychological help and support for patients and family members
To be determined
10:55-10:05 Social care
To be determined
10:05-11:15 Burden of disease
Joseph Muenzer (Germany)
11:15-11:25 Seeking independence (adult patient)
To be determined
11:25-11:35 Patient testimonial
Ana María Mendoza (Spain)
11:35-12:00 Q&A Session
12:00-12:30 QUALITY OF LIFE – TODAY & TOMORROW
Dr. Stewart Rust & patient Jibreel Arshad
12:30-12:45 PRESENTATION 2022 MPS INTERNATIONAL SYMPOSSIUM
12:45-13:00 THANK YOU & FAREWELL
13:00-15:00 LUNCH

Please note:

  • This is a Preliminary Program therefore the speakers, their credentials and title of their sessions are still pending confirmation with each lecturer.
  • The timings of the lectures or sessions might still be subject to changes.