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.

Friday, July 31, 2020

SCIENTIFIC PROGRAM

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
BASICS
Moderator: Dr. Patricia Dickson (USA)
10:45-11:15 Topic: The role of autophagy in lysosomal storage diseases
Dr. Andrea Ballabio (Italy)
11:15-11:45 Topic: Immunogenicity
Dr. Arunabha Ghosh (UK)
11:45-12:15 Topic: CNS Pathology / BBB
To be determined
12:15-12:30 Q&A Session
12:30-14:00 LUNCH
13:00-14:00
Sponsored Lunch Symposium by BIOMARIN
HOT TOPICS & BREAKING NEWS
Moderators: to be determined
14:00-14:20 Topic: Latest animal models for MPS disorders
Dr. Kim Hemsley (Australia)
14:20-14:40 To be determined
14:40-15:00 To be determined
15:00-15:30 Q&A 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
Speakers’ Networking Dinner   Sponsored Dinner Symposium by ABEONA THERAPEUTICS

FAMILY PROGRAM

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) 
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

SCIENTIFIC PROGRAM

07:00-08:30 Sponsored Breakfast Symposium by LYSOGENE
ENZYMATIC THERAPIES
Moderators: Dr. Michael Beck (Germany) & Dr. Mar O’Callaghan (Spain)
08:30-08:55 Topic: Latest Treatments
MD. Paul Harmatz (USA)
08:55-09:20 Topic: Fusion Proteins
Prof. Dr. Roberto Giugliani (Brazil)
09:20-09:45 Topic: Nanoparticles
Dr. Ibane Abasolo (Spain)
09:45-10:15 Oral Poster Presentation
The best Abstracts on the topic will be invited to present
10:15-10:35 Q&A Session
10:35-11:00 COFFEE BREAK
REVIEWING PRESENT & FUTURE OF CELLULAR THERAPY
Moderators: Dr. Christina Lampe (Germany) & Dr. Andria Generoso (Italy)
11:00-11:20 Topic: HSCT transplant, bone marrow transplant
Dr. Jaap Jan Boelens (Netherlands)
11:20-11:40 Topic: Ex vivo therapy MPS II, IIIA, IIIB
Dr. Brian Bigger (UK)
11:40-12:00 Topic: Future of ex vivo gene therapy
Dr. Alessandra Biffi (USA)
12:00-12:30 Q&A Session
12:30-13:00 LUNCH
13:00-14:00 Sponsored Lunch Symposium by SANOFI
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

FAMILY PROGRAM

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

SCIENTIFIC PROGRAM

07:00-08:30 Sponsored Breakfast Symposium by INVENTIVA PHARMA
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
10:45-12:00 ORAL COMMUNICATIONS
Moderators: Dr. Simon Jones (UK) & Dr. Guillem Pintos (Spain)
(The Abstract presentation period will be open from January 16 to March 16. The Scientific Committee will select the best to be presented as oral communications. Early May the Scientific Secretariat will communicate the Abstracts selected for oral presentation. Once the author accepts the presentation, its name and title of the presentation will be included in the program.)
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

FAMILY PROGRAM

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