Please note that this agenda is preliminary and subject to change.
THURSDAY, JUNE 4TH
| Time | Session | Speakers |
|---|---|---|
| 9:00am – 1:00pm | Registration Open | |
| 11:00am-1:00pm | Light Buffet and transfer to congress venue | |
| 1:00pm-2:00pm | Opening Address and Welcome | City of Florence, Tuscany Region. Local authorities and EU |
| 2:00pm-2:45pm | Opening Address | Kim Angel, Maurizio Scarpa, F. Bertoglio |
| 2:45pm-3:15pm | Back 20 Years, Looking to Next 20: A Vision for the MPS Community | Joseph Muenzer |
| 3:15pm-3:45pm | Progress in curing patients: reflecting on 20 years of breakthroughs Improvements in Patient Care and Management Impact of Clinical Trials on Treatment Outcomes | Patty Dickson |
| 3:45pm-4:15pm | Networking Break | |
| 4:15pm-5:00pm | 1. Genetics and pathophysiology of MPS disorders 2. Advancements in diagnostic technologies 3. Open issue on therapeutic approaches for MPS | 1. Robert Giugliani 2. Hsiang-Yu Lin 3. Simon Jones |
| 5:00pm-5:30pm | Keynote presentation: Progress in curing patients: reflecting on 20 years of breakthroughs Improvements in Patient Care and Management Impact of Clinical Trials on Treatment Outcomes Challenges and Solutions in Patient Access to Therapies | Paul Harmatz |
| 6:00pm-8:30pm | Get Together Networking Event |
FRIDAY, JUNE 5TH
| Time | Session | Speakers |
|---|---|---|
| 7:30am-8:30am | Sponsored Workshop | |
| 8:30am-10:00am | TOPIC: ‘Screening 2030 and Beyond: The Evolving Landscape of MPS and related Diseases Newborn Detection | Moderators: Giancarlo La Marca & Mireia del Toro |
| 8:30am-9:00am | Keynote Lecture: Newborn Screening for LSDs: past experience to draft future | Alberto Burlina |
| 9:00am-10:00am | Panel Discussion | Barbara Burton, Paul Harmatz, Robert Giugliani, Michael Gelb Joseph Muenzer, Maria Fueller, John Mitchell, Hsiang-Yu Lin |
| 10:00am-10:30am | Oral Poster Presentations | |
| 10:30am-11:00am | Networking Break and Poster viewing | |
| 11:00am-12:30pm | TOPIC:”Digital Revolution in MPS Care: AI-Driven Diagnosis and Clinical Decision Support” | Moderators: Carlo Dionisi Vici & Florian Lagler |
| 11:00am-11:30am | Keynote Lecture: AI: a new frontier in Rare Disease Early Diagnosis | Alberta Spreafico |
| 11:30am-11:50am | Scientific Lecture: A comparison of Machine Learning Models for MPS Early Diagnosis | Fatma Al Jasmi |
| 11:50am-12:10pm | Scientific Lecture: Machine Learning approaches to assess the impact of rare diseases on patients and healthcare systems | Pierantonio Russo |
| 12:05pm-12:30pm | Keynote Lecture: Regulatory Aspects and Ethics of Artificial Intelligence | |
| 12:30pm-1:00pm | Oral Poster Presentations | |
| 1:00pm-2:00pm | Sponsored Workshop | |
| 2:00pm-2:30pm | TOPIC:”“Advanced Technologies Revolutionizing MPS: Heart, Bone, Eye, and CNS Perspectives” | Moderators: Serena Gasperini & Christina Lampe |
| 2:00pm-2:20pm | Keynote Lecture: New technologies for the understanding of the CSN pathophysiology and paving therapies for LSD | Brian Bigger |
| 2:20pm-2:40pm | Scientific Lecture: Innovative technology for the understanding of pathophysiology and therapy of Heart in MPS | Jacopo Olivotto |
| 2:40pm-3:00pm | Scientific Lecture: Innovative Technology For The Understanding Of Pathophysiology And Therapy Of Skeletal Disorders In MPS | Sunji Tomatsu |
| 3:00pm-3:20pm | Scientific Lecture: Innovative Technology For The Understanding Of Pathophysiology And Therapy Of Eye Disorders In MPS | Giacomo Bacci |
| 3:20pm-3:30pm | Q&A | |
| 3:30pm-4:00pm | Oral Poster Presentations | |
| 4:00pm-5:00pm | Networking Break & Poster Viewing | |
| 5:00pm-6:00pm | Sponsored Workshop |
SATURDAY, JUNE 6TH
| Time | Session | Speakers |
|---|---|---|
| 7:30am-8:30am | Sponsored Workshop | |
| 8:30am-11:00am | TOPIC: The Future of MPS and Related Disorders´ treatment: from gene therapy breakthroughs to regulatory excellence | Moderators: Simona Fecarotta & Nicole Muschol |
| 8:30am-9:00am | Keynote Lecture: Innovative approaches to Cross the Blood Brain Barrier for the treatment of CNS | David Begley & Robert Thorne |
| 9:00am-9:20am | Therapeutic Topics: Gene Therapy where are we targeting? | Alessasndro Fraldi |
| 9:20am-9:40am | Therapeutic Topics: Innovative therapies for LSD: The Promise of Gene Editing, CRISPR, mRNA and Beyond | Natalia Gomez-Ospina |
| 9:40am-10:00am | Therapeutics Topics: Fetal therapy for the treatment of LSDs | Billie Lianoglou |
| 10:00am-10:30am | Oral Poster Presentations | |
| 10:30am-11:00am | Networking Break and Poster Viewing | |
| 11:00am-11:45am | TOPIC: Ethics of Artificial Intelligence | Moderators E. Procopio- M. Solano |
| 11:00am-11:30am | Visionaire Lecture: “One Gene, One Edit, One Cure: The Audacious Promise of Eradicating Mucopolysaccharidose | Chet Whitley |
| 11:30am-11:45am | Q&A | |
| 12:00pm-1:00pm | NOBEL PRICE SPEACH: SYMPOSIUM SPECIAL LECTURE | Moderator: Tim Cox, Kim Angel, Maurizio Scarpa
PROF. SVANTE PÄÄBO |
| 1:00pm-2:00pm | BioMarin Sponsored Workshop: Living with MPS: Honest stories of challenge, resilience and hope | |
| 2:00pm-3:30pm | TOPIC: No patient left behind: Form Florence to the World – building equitable global networks for MPS and related diseases care | Moderators: A. Vellodi-Moeen Al -Sayed |
| 2:00pm-2:30pm | Keynote Lectures: CHALLENGES FOR THE FUTURE: THERAPY ACCESS In The World Access To Therapy Around The World | Chris Hendriks |
| 2:30pm-3:30pm | Panel: Addressing Health Inequities: Strategies For Ensuring Global Equitable Access to MPS and related disorders: toward the creation of a global registry for rare diseases
Panel Discussion: Patients Collaboration And Partnerships: Strengthening Global Networks to Support MPS Patients | Panelist: Robert Giugliani, Marta Solano, Dong-Kzu Jiin, Faith Uzgu, Fatma Al Jasmi, Pranesh Chakraborty , Vu Chin Dung, Yoshikathsu Eto , Meenakshi Bhat , Ben Omran, Jesus Navaro, Ebrahim Golzari Panelist: |
| 3:30pm-4:00pm | Oral Poster Presentations | |
| 4:00pm-5:00pm | Networking Break & Poster Viewing | |
| 5:00pm-6:00pm | Sponsored Workshop | |
| 6:30pm-8:30pm | Networking Dinner |
SUNDAY, JUNE 7TH
| Time | Session | Speakers |
|---|---|---|
| 7:30am-8:30am | Sponsored Workshop | |
| 8:30am-9:30am | TOPIC: Late breaking news and new frontiers | Moderators: F. Deodato-A. Dardis |
| 9:30am-9:45am | Q&A | |
| 9:45am-10:15am | Awards Presentation | |
| 10:45am-11:15am | Networking Break & Poster Viewing | |
| 11:15am-1:00pm | PANEL DISCUSSION: Vision for the Next 20 Years: Unified Global Network, Future Treatments, Long-Term Monitoring Family and Scientific selected speakers to identify a program to be pursued under the IMPSN leadership at global level THE FUTURE OF MPS | Panelists: Tim Cox, Chris Hendriks, John Mitchell, Robert Giugliani, Michal Inbar-Feigenberg , Matthew Ellinwood, Brian Bigger, T.M.Keong, Joseph Muenzer, Family representatives, Adrian Toggenburger |
| 1:00pm-2:00pm | Closing Up – summary of the meeting and adjourn to 2028 |